Barriers to gene therapy, understanding the concerns people with haemophilia have: an exigency sub-study
Background Gene therapy has the potential to offer people with haemophilia (PwH) a life free from bleeding and the burden posed by current treatment regimens. To date, gene therapy has only been available in clinical trial settings, to PwH without pre-existing or historical factor inhibitors, signif...
Gespeichert in:
| Veröffentlicht in: | Orphanet journal of rare diseases Jg. 19; H. 1; S. 59 - 12 |
|---|---|
| Hauptverfasser: | , , , |
| Format: | Journal Article |
| Sprache: | Englisch |
| Veröffentlicht: |
London
BioMed Central
10.02.2024
BioMed Central Ltd Springer Nature B.V BMC |
| Schlagworte: | |
| ISSN: | 1750-1172, 1750-1172 |
| Online-Zugang: | Volltext |
| Tags: |
Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!
|
| Abstract | Background
Gene therapy has the potential to offer people with haemophilia (PwH) a life free from bleeding and the burden posed by current treatment regimens. To date, gene therapy has only been available in clinical trial settings, to PwH without pre-existing or historical factor inhibitors, significant concomitant liver damage or pre-existing neutralising antibodies to the adeno-associated viruses used to deliver the therapy. Thus, most PwH treated at centres not currently involved in gene therapy trials, either as a referral/follow-up centre or as a dosing centre, have been unable to access the therapy. This Exigency sub-study aims to gain a greater understanding of the opinions of PwH in the United Kingdom who have not had access to gene therapy: asking what they understand, what concerns they have, and whether they perceive any barriers preventing their access to gene therapy.
Results
Twenty-three PwH were approached; 14 consented, and one withdrew prior to interview. The mean age of the participants was 35.7 years (range 25–74 years). Eleven had haemophilia A and two haemophilia B. Two were treated with standard half-life factor products, five with extended half-life products, five with a FVIII mimetic and one with a clinical trial product. One family member (a participant's partner) was also interviewed. The participants identified four barriers to gene therapy: concerns about the process of gene therapy (Expectations), uncertainty about the results (outcomes), (Access) to treatment, and a lack of understanding about gene therapy (education).
Conclusions
This Exigency study subgroup sees gene therapy as a positive treatment development that promises an improved quality of life. For this participant group, four issues impact their decision to undergo gene therapy. If the promise of gene therapy is to be realised, these barriers need to be acknowledged and addressed by healthcare professionals, patient organisations, and gene therapy providers. |
|---|---|
| AbstractList | Gene therapy has the potential to offer people with haemophilia (PwH) a life free from bleeding and the burden posed by current treatment regimens. To date, gene therapy has only been available in clinical trial settings, to PwH without pre-existing or historical factor inhibitors, significant concomitant liver damage or pre-existing neutralising antibodies to the adeno-associated viruses used to deliver the therapy. Thus, most PwH treated at centres not currently involved in gene therapy trials, either as a referral/follow-up centre or as a dosing centre, have been unable to access the therapy. This Exigency sub-study aims to gain a greater understanding of the opinions of PwH in the United Kingdom who have not had access to gene therapy: asking what they understand, what concerns they have, and whether they perceive any barriers preventing their access to gene therapy. Twenty-three PwH were approached; 14 consented, and one withdrew prior to interview. The mean age of the participants was 35.7 years (range 25-74 years). Eleven had haemophilia A and two haemophilia B. Two were treated with standard half-life factor products, five with extended half-life products, five with a FVIII mimetic and one with a clinical trial product. One family member (a participant's partner) was also interviewed. The participants identified four barriers to gene therapy: concerns about the process of gene therapy (Expectations), uncertainty about the results (outcomes), (Access) to treatment, and a lack of understanding about gene therapy (education). This Exigency study subgroup sees gene therapy as a positive treatment development that promises an improved quality of life. For this participant group, four issues impact their decision to undergo gene therapy. If the promise of gene therapy is to be realised, these barriers need to be acknowledged and addressed by healthcare professionals, patient organisations, and gene therapy providers. Background Gene therapy has the potential to offer people with haemophilia (PwH) a life free from bleeding and the burden posed by current treatment regimens. To date, gene therapy has only been available in clinical trial settings, to PwH without pre-existing or historical factor inhibitors, significant concomitant liver damage or pre-existing neutralising antibodies to the adeno-associated viruses used to deliver the therapy. Thus, most PwH treated at centres not currently involved in gene therapy trials, either as a referral/follow-up centre or as a dosing centre, have been unable to access the therapy. This Exigency sub-study aims to gain a greater understanding of the opinions of PwH in the United Kingdom who have not had access to gene therapy: asking what they understand, what concerns they have, and whether they perceive any barriers preventing their access to gene therapy. Results Twenty-three PwH were approached; 14 consented, and one withdrew prior to interview. The mean age of the participants was 35.7 years (range 25–74 years). Eleven had haemophilia A and two haemophilia B. Two were treated with standard half-life factor products, five with extended half-life products, five with a FVIII mimetic and one with a clinical trial product. One family member (a participant's partner) was also interviewed. The participants identified four barriers to gene therapy: concerns about the process of gene therapy (Expectations), uncertainty about the results (outcomes), (Access) to treatment, and a lack of understanding about gene therapy (education). Conclusions This Exigency study subgroup sees gene therapy as a positive treatment development that promises an improved quality of life. For this participant group, four issues impact their decision to undergo gene therapy. If the promise of gene therapy is to be realised, these barriers need to be acknowledged and addressed by healthcare professionals, patient organisations, and gene therapy providers. Gene therapy has the potential to offer people with haemophilia (PwH) a life free from bleeding and the burden posed by current treatment regimens. To date, gene therapy has only been available in clinical trial settings, to PwH without pre-existing or historical factor inhibitors, significant concomitant liver damage or pre-existing neutralising antibodies to the adeno-associated viruses used to deliver the therapy. Thus, most PwH treated at centres not currently involved in gene therapy trials, either as a referral/follow-up centre or as a dosing centre, have been unable to access the therapy. This Exigency sub-study aims to gain a greater understanding of the opinions of PwH in the United Kingdom who have not had access to gene therapy: asking what they understand, what concerns they have, and whether they perceive any barriers preventing their access to gene therapy. Twenty-three PwH were approached; 14 consented, and one withdrew prior to interview. The mean age of the participants was 35.7 years (range 25-74 years). Eleven had haemophilia A and two haemophilia B. Two were treated with standard half-life factor products, five with extended half-life products, five with a FVIII mimetic and one with a clinical trial product. One family member (a participant's partner) was also interviewed. The participants identified four barriers to gene therapy: concerns about the process of gene therapy (Expectations), uncertainty about the results (outcomes), (Access) to treatment, and a lack of understanding about gene therapy (education). This Exigency study subgroup sees gene therapy as a positive treatment development that promises an improved quality of life. For this participant group, four issues impact their decision to undergo gene therapy. If the promise of gene therapy is to be realised, these barriers need to be acknowledged and addressed by healthcare professionals, patient organisations, and gene therapy providers. Background Gene therapy has the potential to offer people with haemophilia (PwH) a life free from bleeding and the burden posed by current treatment regimens. To date, gene therapy has only been available in clinical trial settings, to PwH without pre-existing or historical factor inhibitors, significant concomitant liver damage or pre-existing neutralising antibodies to the adeno-associated viruses used to deliver the therapy. Thus, most PwH treated at centres not currently involved in gene therapy trials, either as a referral/follow-up centre or as a dosing centre, have been unable to access the therapy. This Exigency sub-study aims to gain a greater understanding of the opinions of PwH in the United Kingdom who have not had access to gene therapy: asking what they understand, what concerns they have, and whether they perceive any barriers preventing their access to gene therapy. Results Twenty-three PwH were approached; 14 consented, and one withdrew prior to interview. The mean age of the participants was 35.7 years (range 25-74 years). Eleven had haemophilia A and two haemophilia B. Two were treated with standard half-life factor products, five with extended half-life products, five with a FVIII mimetic and one with a clinical trial product. One family member (a participant's partner) was also interviewed. The participants identified four barriers to gene therapy: concerns about the process of gene therapy (Expectations), uncertainty about the results (outcomes), (Access) to treatment, and a lack of understanding about gene therapy (education). Conclusions This Exigency study subgroup sees gene therapy as a positive treatment development that promises an improved quality of life. For this participant group, four issues impact their decision to undergo gene therapy. If the promise of gene therapy is to be realised, these barriers need to be acknowledged and addressed by healthcare professionals, patient organisations, and gene therapy providers. Keywords: Haemophilia, Gene therapy, Shared decision-making, Outcomes, Quality of life BackgroundGene therapy has the potential to offer people with haemophilia (PwH) a life free from bleeding and the burden posed by current treatment regimens. To date, gene therapy has only been available in clinical trial settings, to PwH without pre-existing or historical factor inhibitors, significant concomitant liver damage or pre-existing neutralising antibodies to the adeno-associated viruses used to deliver the therapy. Thus, most PwH treated at centres not currently involved in gene therapy trials, either as a referral/follow-up centre or as a dosing centre, have been unable to access the therapy. This Exigency sub-study aims to gain a greater understanding of the opinions of PwH in the United Kingdom who have not had access to gene therapy: asking what they understand, what concerns they have, and whether they perceive any barriers preventing their access to gene therapy.ResultsTwenty-three PwH were approached; 14 consented, and one withdrew prior to interview. The mean age of the participants was 35.7 years (range 25–74 years). Eleven had haemophilia A and two haemophilia B. Two were treated with standard half-life factor products, five with extended half-life products, five with a FVIII mimetic and one with a clinical trial product. One family member (a participant's partner) was also interviewed. The participants identified four barriers to gene therapy: concerns about the process of gene therapy (Expectations), uncertainty about the results (outcomes), (Access) to treatment, and a lack of understanding about gene therapy (education).ConclusionsThis Exigency study subgroup sees gene therapy as a positive treatment development that promises an improved quality of life. For this participant group, four issues impact their decision to undergo gene therapy. If the promise of gene therapy is to be realised, these barriers need to be acknowledged and addressed by healthcare professionals, patient organisations, and gene therapy providers. Abstract Background Gene therapy has the potential to offer people with haemophilia (PwH) a life free from bleeding and the burden posed by current treatment regimens. To date, gene therapy has only been available in clinical trial settings, to PwH without pre-existing or historical factor inhibitors, significant concomitant liver damage or pre-existing neutralising antibodies to the adeno-associated viruses used to deliver the therapy. Thus, most PwH treated at centres not currently involved in gene therapy trials, either as a referral/follow-up centre or as a dosing centre, have been unable to access the therapy. This Exigency sub-study aims to gain a greater understanding of the opinions of PwH in the United Kingdom who have not had access to gene therapy: asking what they understand, what concerns they have, and whether they perceive any barriers preventing their access to gene therapy. Results Twenty-three PwH were approached; 14 consented, and one withdrew prior to interview. The mean age of the participants was 35.7 years (range 25–74 years). Eleven had haemophilia A and two haemophilia B. Two were treated with standard half-life factor products, five with extended half-life products, five with a FVIII mimetic and one with a clinical trial product. One family member (a participant's partner) was also interviewed. The participants identified four barriers to gene therapy: concerns about the process of gene therapy (Expectations), uncertainty about the results (outcomes), (Access) to treatment, and a lack of understanding about gene therapy (education). Conclusions This Exigency study subgroup sees gene therapy as a positive treatment development that promises an improved quality of life. For this participant group, four issues impact their decision to undergo gene therapy. If the promise of gene therapy is to be realised, these barriers need to be acknowledged and addressed by healthcare professionals, patient organisations, and gene therapy providers. Gene therapy has the potential to offer people with haemophilia (PwH) a life free from bleeding and the burden posed by current treatment regimens. To date, gene therapy has only been available in clinical trial settings, to PwH without pre-existing or historical factor inhibitors, significant concomitant liver damage or pre-existing neutralising antibodies to the adeno-associated viruses used to deliver the therapy. Thus, most PwH treated at centres not currently involved in gene therapy trials, either as a referral/follow-up centre or as a dosing centre, have been unable to access the therapy. This Exigency sub-study aims to gain a greater understanding of the opinions of PwH in the United Kingdom who have not had access to gene therapy: asking what they understand, what concerns they have, and whether they perceive any barriers preventing their access to gene therapy.BACKGROUNDGene therapy has the potential to offer people with haemophilia (PwH) a life free from bleeding and the burden posed by current treatment regimens. To date, gene therapy has only been available in clinical trial settings, to PwH without pre-existing or historical factor inhibitors, significant concomitant liver damage or pre-existing neutralising antibodies to the adeno-associated viruses used to deliver the therapy. Thus, most PwH treated at centres not currently involved in gene therapy trials, either as a referral/follow-up centre or as a dosing centre, have been unable to access the therapy. This Exigency sub-study aims to gain a greater understanding of the opinions of PwH in the United Kingdom who have not had access to gene therapy: asking what they understand, what concerns they have, and whether they perceive any barriers preventing their access to gene therapy.Twenty-three PwH were approached; 14 consented, and one withdrew prior to interview. The mean age of the participants was 35.7 years (range 25-74 years). Eleven had haemophilia A and two haemophilia B. Two were treated with standard half-life factor products, five with extended half-life products, five with a FVIII mimetic and one with a clinical trial product. One family member (a participant's partner) was also interviewed. The participants identified four barriers to gene therapy: concerns about the process of gene therapy (Expectations), uncertainty about the results (outcomes), (Access) to treatment, and a lack of understanding about gene therapy (education).RESULTSTwenty-three PwH were approached; 14 consented, and one withdrew prior to interview. The mean age of the participants was 35.7 years (range 25-74 years). Eleven had haemophilia A and two haemophilia B. Two were treated with standard half-life factor products, five with extended half-life products, five with a FVIII mimetic and one with a clinical trial product. One family member (a participant's partner) was also interviewed. The participants identified four barriers to gene therapy: concerns about the process of gene therapy (Expectations), uncertainty about the results (outcomes), (Access) to treatment, and a lack of understanding about gene therapy (education).This Exigency study subgroup sees gene therapy as a positive treatment development that promises an improved quality of life. For this participant group, four issues impact their decision to undergo gene therapy. If the promise of gene therapy is to be realised, these barriers need to be acknowledged and addressed by healthcare professionals, patient organisations, and gene therapy providers.CONCLUSIONSThis Exigency study subgroup sees gene therapy as a positive treatment development that promises an improved quality of life. For this participant group, four issues impact their decision to undergo gene therapy. If the promise of gene therapy is to be realised, these barriers need to be acknowledged and addressed by healthcare professionals, patient organisations, and gene therapy providers. |
| ArticleNumber | 59 |
| Audience | Academic |
| Author | Khair, Kate Jenner, Kathryn Holland, Michael Fletcher, Simon |
| Author_xml | – sequence: 1 givenname: Simon orcidid: 0000-0001-9018-6176 surname: Fletcher fullname: Fletcher, Simon email: sfletcher26@aol.com organization: Haemnet, Oxford Haemophilia and Thrombosis Centre, Oxford University Hospitals NHS Foundation Trust – sequence: 2 givenname: Kathryn orcidid: 0000-0002-2704-0606 surname: Jenner fullname: Jenner, Kathryn organization: Haemnet – sequence: 3 givenname: Michael orcidid: 0000-0002-9173-4100 surname: Holland fullname: Holland, Michael organization: Haemnet – sequence: 4 givenname: Kate orcidid: 0000-0003-2001-5958 surname: Khair fullname: Khair, Kate organization: Haemnet |
| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/38341591$$D View this record in MEDLINE/PubMed |
| BookMark | eNp9Uttq3DAUNCWlSbb9gT4UQ19aqFNdbEvuS0lDLwuBQi_PQpaObQWvtJHkNPv3lXeTJhtKEEiHo5k5aDTH2YF1FrLsJUYnGPP6fcAUEVogUhaIopoX5El2hFmFCowZObhXH2bHIVwgVFYU8WfZIeW0xFWDj7Lhk_TegA95dHkPFvI4gJfrzbt8sjr1o7Ta2H5u58pZBd6GfA1uPUL-x8QhHySs3Howo5GpvoIPubQ5XJskpjZ5mNoixElvnmdPOzkGeHFzLrLfXz7_OvtWnH__ujw7PS9UXZJYyFbVkDagmBLZlJhWutYcM02aRjKulGIALaoY4BZYy3Sjy6auOGjQGhBdZMudrnbyQqy9WUm_EU4asW043wvpo1EjCEI7zEvWKt3UZRoqy5rRpuOyw9BxXiWtjzut9dSuQCuw0ctxT3T_xppB9O5KYMSrBmGaFN7cKHh3OUGIYmWCgnGUFtwUBGlIhSgt6Tzs9QPohZu8TV5tUXXN6rq8Q_UyvcDYzqXBahYVp4wTghDHswkn_0GlpWFl0i9CZ1J_j_B2j5AwEa5jL6cQxPLnj33sq_uu_LPjNlQJwHcA5V0IHjqhTJTRuNkkMyZ3xJxfscuvSPkV2_ymD1lk5AH1Vv1REt2RQgLbHvydc4-w_gIgPgGv |
| CitedBy_id | crossref_primary_10_2478_jhp_2024_0012 crossref_primary_10_1007_s44337_025_00376_9 crossref_primary_10_3390_biom15030378 crossref_primary_10_1111_hae_70008 crossref_primary_10_1146_annurev_genom_021623_104458 crossref_primary_10_2478_jhp_2025_0001 |
| Cites_doi | 10.1182/blood-2018-99-118050 10.1177/1049732318788379 10.4103/2249-4863.161306 10.2478/jhp-2023-0009 10.1038/d41586-022-04327-7 10.1111/hae.14378 10.1016/S0277-9536(96)00221-3 10.2147/PPA.S205819 10.1007/s10728-008-0108-6 10.1056/NEJMoa1108046 10.1177/1609406920937875 10.1056/NEJMoa1708538 10.2147/PPA.S355627 10.1186/s13023-022-02256-2 10.1016/j.jtha.2022.12.027 10.1111/hae.12495 10.1007/s13337-021-00715-1 10.2478/jhp-2022-0021 10.1371/journal.pone.0232076 10.1111/hae.14046 10.1182/blood-2018-07-820720 10.1056/NEJMoa1708483 10.1111/hae.14774 10.17225/jhp00174 10.1176/appi.ps.201400307 10.1182/blood-2018-99-118256 10.1001/virtualmentor.2012.14.7.oped2-1207 10.1111/hae.14190 10.1186/s12913-019-4649-1 10.1056/NEJMoa2211644 10.1056/NEJMoa2104205 10.2147/PPA.S239810 10.1111/j.1365-2648.2009.05250.x 10.1177/1609406919874596 10.1111/j.1365-2648.2006.03721.x 10.1111/hae.14214 10.1136/bmjopen-2021-060351 10.17225/jhp/00178 10.1016/j.blre.2021.100890 10.2478/jhp-2021-0019 10.1007/s13337-020-00590-2 10.1111/hae.14420 10.1136/bmj.open-2019-031763 |
| ContentType | Journal Article |
| Copyright | The Author(s) 2024 2024. The Author(s). COPYRIGHT 2024 BioMed Central Ltd. 2024. This work is licensed under http://creativecommons.org/licenses/by/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License. |
| Copyright_xml | – notice: The Author(s) 2024 – notice: 2024. The Author(s). – notice: COPYRIGHT 2024 BioMed Central Ltd. – notice: 2024. This work is licensed under http://creativecommons.org/licenses/by/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License. |
| DBID | C6C AAYXX CITATION CGR CUY CVF ECM EIF NPM ISR 3V. 7T5 7X7 7XB 88E 8FI 8FJ 8FK ABUWG AFKRA AN0 AZQEC BENPR CCPQU COVID DWQXO FYUFA GHDGH H94 K9. M0S M1P PHGZM PHGZT PIMPY PJZUB PKEHL PPXIY PQEST PQQKQ PQUKI PRINS 7X8 5PM DOA |
| DOI | 10.1186/s13023-024-03068-2 |
| DatabaseName | Springer Nature OA Free Journals CrossRef Medline MEDLINE MEDLINE (Ovid) MEDLINE MEDLINE PubMed Gale In Context: Science ProQuest Central (Corporate) Immunology Abstracts Health & Medical Collection ProQuest Central (purchase pre-March 2016) Medical Database (Alumni Edition) Hospital Premium Collection Hospital Premium Collection (Alumni Edition) ProQuest Central (Alumni) (purchase pre-March 2016) ProQuest Central (Alumni) ProQuest Central UK/Ireland British Nursing Database ProQuest Central Essentials ProQuest Central ProQuest One Community College Coronavirus Research Database ProQuest Central Health Research Premium Collection Health Research Premium Collection (Alumni) AIDS and Cancer Research Abstracts ProQuest Health & Medical Complete (Alumni) Health & Medical Collection (Alumni Edition) PML(ProQuest Medical Library) ProQuest Central Premium ProQuest One Academic (New) ProQuest Publicly Available Content ProQuest Health & Medical Research Collection ProQuest One Academic Middle East (New) ProQuest One Health & Nursing ProQuest One Academic Eastern Edition (DO NOT USE) ProQuest One Academic (retired) ProQuest One Academic UKI Edition ProQuest Central China MEDLINE - Academic PubMed Central (Full Participant titles) DOAJ Open Access Full Text |
| DatabaseTitle | CrossRef MEDLINE Medline Complete MEDLINE with Full Text PubMed MEDLINE (Ovid) Publicly Available Content Database ProQuest One Academic Middle East (New) ProQuest Central Essentials ProQuest Health & Medical Complete (Alumni) ProQuest Central (Alumni Edition) ProQuest One Community College ProQuest One Health & Nursing ProQuest Central China ProQuest Central ProQuest Health & Medical Research Collection Health Research Premium Collection Health and Medicine Complete (Alumni Edition) ProQuest Central Korea Health & Medical Research Collection AIDS and Cancer Research Abstracts ProQuest Central (New) ProQuest Medical Library (Alumni) ProQuest One Academic Eastern Edition British Nursing Index with Full Text Coronavirus Research Database ProQuest Hospital Collection Health Research Premium Collection (Alumni) ProQuest Hospital Collection (Alumni) ProQuest Health & Medical Complete ProQuest Medical Library ProQuest One Academic UKI Edition Immunology Abstracts ProQuest One Academic ProQuest One Academic (New) ProQuest Central (Alumni) MEDLINE - Academic |
| DatabaseTitleList | MEDLINE Publicly Available Content Database MEDLINE - Academic |
| Database_xml | – sequence: 1 dbid: DOA name: DOAJ Open Access Full Text url: https://www.doaj.org/ sourceTypes: Open Website – sequence: 2 dbid: NPM name: PubMed url: http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=PubMed sourceTypes: Index Database – sequence: 3 dbid: PIMPY name: Publicly Available Content Database url: http://search.proquest.com/publiccontent sourceTypes: Aggregation Database |
| DeliveryMethod | fulltext_linktorsrc |
| Discipline | Medicine |
| EISSN | 1750-1172 |
| EndPage | 12 |
| ExternalDocumentID | oai_doaj_org_article_23f1847bcd964eaba46739f8af1ef885 PMC10859013 A782200810 38341591 10_1186_s13023_024_03068_2 |
| Genre | Journal Article |
| GeographicLocations | United Kingdom United Kingdom--UK |
| GeographicLocations_xml | – name: United Kingdom – name: United Kingdom--UK |
| GrantInformation_xml | – fundername: uniQure grantid: 2-061-003 - Haemophilia B - Late development |
| GroupedDBID | --- 0R~ 123 29N 2WC 53G 5VS 7X7 88E 8FI 8FJ AAFWJ AAJSJ AASML AAWTL ABDBF ABUWG ACGFO ACGFS ACIHN ACPRK ACUHS ADBBV ADRAZ ADUKV AEAQA AENEX AFKRA AFPKN AHBYD AHMBA AHYZX ALMA_UNASSIGNED_HOLDINGS AMKLP AMTXH AN0 AOIJS BAPOH BAWUL BCNDV BENPR BFQNJ BMC BNQBC BPHCQ BVXVI C6C CCPQU CS3 DIK DU5 E3Z EBD EBLON EBS EMOBN ESX F5P FYUFA GROUPED_DOAJ GX1 HMCUK HYE IAO IHR INH INR ISR ITC KQ8 M1P M48 MK0 M~E O5R O5S OK1 OVT P2P PGMZT PHGZM PHGZT PIMPY PJZUB PPXIY PQQKQ PROAC PSQYO PUEGO RBZ RNS ROL RPM RSV SMD SOJ SV3 TR2 TUS UKHRP WOQ WOW ~8M AAYXX AFFHD CITATION ALIPV CGR CUY CVF ECM EIF NPM 3V. 7T5 7XB 8FK AZQEC COVID DWQXO H94 K9. PKEHL PQEST PQUKI PRINS 7X8 5PM |
| ID | FETCH-LOGICAL-c642t-abc6eabce3132a94135d6d817d299a78ccc7eeb057e1be7b7d9d49658ededde03 |
| IEDL.DBID | DOA |
| ISICitedReferencesCount | 3 |
| ISICitedReferencesURI | http://www.webofscience.com/api/gateway?GWVersion=2&SrcApp=Summon&SrcAuth=ProQuest&DestLinkType=CitingArticles&DestApp=WOS_CPL&KeyUT=001161263600001&url=https%3A%2F%2Fcvtisr.summon.serialssolutions.com%2F%23%21%2Fsearch%3Fho%3Df%26include.ft.matches%3Dt%26l%3Dnull%26q%3D |
| ISSN | 1750-1172 |
| IngestDate | Fri Oct 03 12:53:16 EDT 2025 Tue Nov 04 02:05:46 EST 2025 Sun Nov 09 09:29:24 EST 2025 Mon Oct 20 02:56:37 EDT 2025 Tue Nov 11 11:15:03 EST 2025 Tue Nov 04 18:33:00 EST 2025 Wed Nov 26 11:20:46 EST 2025 Mon Jul 21 05:59:48 EDT 2025 Sat Nov 29 03:40:48 EST 2025 Tue Nov 18 20:47:26 EST 2025 Sat Sep 06 07:29:01 EDT 2025 |
| IsDoiOpenAccess | true |
| IsOpenAccess | true |
| IsPeerReviewed | true |
| IsScholarly | true |
| Issue | 1 |
| Keywords | Haemophilia Gene therapy Outcomes Shared decision-making Quality of life |
| Language | English |
| License | 2024. The Author(s). Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated in a credit line to the data. |
| LinkModel | DirectLink |
| MergedId | FETCHMERGED-LOGICAL-c642t-abc6eabce3132a94135d6d817d299a78ccc7eeb057e1be7b7d9d49658ededde03 |
| Notes | ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 14 content type line 23 |
| ORCID | 0000-0001-9018-6176 0000-0002-2704-0606 0000-0002-9173-4100 0000-0003-2001-5958 |
| OpenAccessLink | https://doaj.org/article/23f1847bcd964eaba46739f8af1ef885 |
| PMID | 38341591 |
| PQID | 2925667664 |
| PQPubID | 76088 |
| PageCount | 12 |
| ParticipantIDs | doaj_primary_oai_doaj_org_article_23f1847bcd964eaba46739f8af1ef885 pubmedcentral_primary_oai_pubmedcentral_nih_gov_10859013 proquest_miscellaneous_2925033435 proquest_journals_2925667664 gale_infotracmisc_A782200810 gale_infotracacademiconefile_A782200810 gale_incontextgauss_ISR_A782200810 pubmed_primary_38341591 crossref_citationtrail_10_1186_s13023_024_03068_2 crossref_primary_10_1186_s13023_024_03068_2 springer_journals_10_1186_s13023_024_03068_2 |
| PublicationCentury | 2000 |
| PublicationDate | 2024-02-10 |
| PublicationDateYYYYMMDD | 2024-02-10 |
| PublicationDate_xml | – month: 02 year: 2024 text: 2024-02-10 day: 10 |
| PublicationDecade | 2020 |
| PublicationPlace | London |
| PublicationPlace_xml | – name: London – name: England |
| PublicationTitle | Orphanet journal of rare diseases |
| PublicationTitleAbbrev | Orphanet J Rare Dis |
| PublicationTitleAlternate | Orphanet J Rare Dis |
| PublicationYear | 2024 |
| Publisher | BioMed Central BioMed Central Ltd Springer Nature B.V BMC |
| Publisher_xml | – name: BioMed Central – name: BioMed Central Ltd – name: Springer Nature B.V – name: BMC |
| References | L Sandman (3068_CR30) 2010; 18 S Akhter (3068_CR21) 2020; 31 S Fletcher (3068_CR13) 2022; 17 3068_CR25 C Hermans (3068_CR35) 2022; 52 P Chowdary (3068_CR24) 2018; 132 S Fletcher (3068_CR39) 2023; 10 L Zimmer (3068_CR45) 2006; 53 E Aradom (3068_CR12) 2021; 8 GQ Perin (3068_CR5) 2018; 33 D Noone (3068_CR26) 2022; 9 LA Valentino (3068_CR32) 2021; 8 E van Overbeeke (3068_CR15) 2020; 27 T Waldron (3068_CR28) 2020; 20 W Miesbach (3068_CR9) 2021; 27 SW Pipe (3068_CR20) 2023; 388 3068_CR8 LA George (3068_CR19) 2017; 377 L Bailo (3068_CR38) 2019; 13 3068_CR6 M Naddaf (3068_CR7) 2022; 612 LA George (3068_CR2) 2017; 377 L Leung (3068_CR43) 2015; 4 AC Nathwani (3068_CR1) 2011; 365 LA George (3068_CR3) 2021; 385 A Athale (3068_CR29) 2014; 20 S Fletcher (3068_CR17) 2022; 12 L Baas (3068_CR47) 2023 S Fletcher (3068_CR50) 2021; 8 S Fletcher (3068_CR14) 2023 C Charles (3068_CR27) 1997; 44 S Rangarajan (3068_CR4) 2017; 377 W Meisbach (3068_CR11) 2020; 14 BB Basnet (3068_CR22) 2021; 32 G Guest (3068_CR18) 2020; 15 L Carminati (3068_CR44) 2018; 28 S Fletcher (3068_CR16) 2021; 1 H Bomhof-Roordink (3068_CR31) 2019; 9 J Hamann (3068_CR36) 2014; 65 3068_CR41 B Lobe (3068_CR49) 2020 3068_CR42 M Wang (3068_CR34) 2022; 16 3068_CR40 MM Archibald (3068_CR48) 2019; 18 AC Nathwani (3068_CR23) 2018; 132 L Woollard (3068_CR33) 2021; 27 D Finfgeld-Connett (3068_CR46) 2010; 66 3068_CR10 BC Drolet (3068_CR37) 2012; 4 |
| References_xml | – volume: 132 start-page: 631 year: 2018 ident: 3068_CR24 publication-title: Blood doi: 10.1182/blood-2018-99-118050 – volume: 28 start-page: 2094 issue: 13 year: 2018 ident: 3068_CR44 publication-title: Qual Health Res doi: 10.1177/1049732318788379 – volume: 4 start-page: 324 issue: 3 year: 2015 ident: 3068_CR43 publication-title: J Family Med Prim Care doi: 10.4103/2249-4863.161306 – volume: 10 start-page: 62 issue: 1 year: 2023 ident: 3068_CR39 publication-title: J Haem Pract doi: 10.2478/jhp-2023-0009 – volume: 612 start-page: 388 year: 2022 ident: 3068_CR7 publication-title: Nature. doi: 10.1038/d41586-022-04327-7 – volume: 1 start-page: 9 year: 2021 ident: 3068_CR16 publication-title: Haemophilia. doi: 10.1111/hae.14378 – volume: 44 start-page: 681 issue: 5 year: 1997 ident: 3068_CR27 publication-title: Soc Sci Med doi: 10.1016/S0277-9536(96)00221-3 – volume: 13 start-page: 2225 year: 2019 ident: 3068_CR38 publication-title: Patient Prefer Adherence doi: 10.2147/PPA.S205819 – volume: 18 start-page: 60 issue: 1 year: 2010 ident: 3068_CR30 publication-title: Health Care Anal doi: 10.1007/s10728-008-0108-6 – volume: 365 start-page: 2357 year: 2011 ident: 3068_CR1 publication-title: N Engl J Med. doi: 10.1056/NEJMoa1108046 – year: 2020 ident: 3068_CR49 publication-title: Int J Qual Methods doi: 10.1177/1609406920937875 – volume: 377 start-page: 2215 issue: 23 year: 2017 ident: 3068_CR2 publication-title: N Engl J Med. doi: 10.1056/NEJMoa1708538 – volume: 16 start-page: 1439 year: 2022 ident: 3068_CR34 publication-title: Patient Prefer Adherence doi: 10.2147/PPA.S355627 – volume: 17 start-page: 155 year: 2022 ident: 3068_CR13 publication-title: Orphanet J Rare Dis doi: 10.1186/s13023-022-02256-2 – year: 2023 ident: 3068_CR47 publication-title: J Thromb Haemost doi: 10.1016/j.jtha.2022.12.027 – volume: 20 start-page: 800 issue: 6 year: 2014 ident: 3068_CR29 publication-title: Haemophilia doi: 10.1111/hae.12495 – ident: 3068_CR41 – volume: 32 start-page: 770 year: 2021 ident: 3068_CR22 publication-title: VirusDis. doi: 10.1007/s13337-021-00715-1 – volume: 9 start-page: 156 issue: 1 year: 2022 ident: 3068_CR26 publication-title: J Haem Pract doi: 10.2478/jhp-2022-0021 – volume: 15 start-page: e0232076 issue: 5 year: 2020 ident: 3068_CR18 publication-title: PLoS ONE doi: 10.1371/journal.pone.0232076 – ident: 3068_CR25 doi: 10.1111/hae.14046 – volume: 33 start-page: 407 year: 2018 ident: 3068_CR5 publication-title: Blood. doi: 10.1182/blood-2018-07-820720 – volume: 377 start-page: 2519 year: 2017 ident: 3068_CR4 publication-title: N Engl J Med doi: 10.1056/NEJMoa1708483 – year: 2023 ident: 3068_CR14 publication-title: Haemophilia doi: 10.1111/hae.14774 – volume: 8 start-page: 32 year: 2021 ident: 3068_CR12 publication-title: Journal of Haemophilia Practice. doi: 10.17225/jhp00174 – ident: 3068_CR6 – volume: 65 start-page: 1483 year: 2014 ident: 3068_CR36 publication-title: Psychiatr Serv doi: 10.1176/appi.ps.201400307 – ident: 3068_CR42 – volume: 132 start-page: 489 year: 2018 ident: 3068_CR23 publication-title: Blood doi: 10.1182/blood-2018-99-118256 – volume: 4 start-page: 582 year: 2012 ident: 3068_CR37 publication-title: AMA J Ethics doi: 10.1001/virtualmentor.2012.14.7.oped2-1207 – volume: 27 start-page: 129 year: 2020 ident: 3068_CR15 publication-title: Haemophilia doi: 10.1111/hae.14190 – volume: 20 start-page: 59 year: 2020 ident: 3068_CR28 publication-title: BMC Health Serv Res. doi: 10.1186/s12913-019-4649-1 – ident: 3068_CR10 – volume: 377 start-page: 2215 issue: 23 year: 2017 ident: 3068_CR19 publication-title: N Engl J Med doi: 10.1056/NEJMoa1708538 – volume: 388 start-page: 706 year: 2023 ident: 3068_CR20 publication-title: N Engl J Med. doi: 10.1056/NEJMoa2211644 – volume: 385 start-page: 1961 year: 2021 ident: 3068_CR3 publication-title: N Engl J Med doi: 10.1056/NEJMoa2104205 – volume: 14 start-page: 767 year: 2020 ident: 3068_CR11 publication-title: Patient Prefer Adherence doi: 10.2147/PPA.S239810 – volume: 66 start-page: 246 year: 2010 ident: 3068_CR46 publication-title: J Adv Nurs doi: 10.1111/j.1365-2648.2009.05250.x – volume: 18 start-page: 1 year: 2019 ident: 3068_CR48 publication-title: Int J Qual Methods doi: 10.1177/1609406919874596 – volume: 53 start-page: 311 year: 2006 ident: 3068_CR45 publication-title: J Adv Nurs doi: 10.1111/j.1365-2648.2006.03721.x – volume: 27 start-page: e302 issue: 2 year: 2021 ident: 3068_CR33 publication-title: Haemophilia doi: 10.1111/hae.14214 – volume: 12 start-page: e060351 year: 2022 ident: 3068_CR17 publication-title: BMJ Open doi: 10.1136/bmjopen-2021-060351 – volume: 8 start-page: 69 issue: 1 year: 2021 ident: 3068_CR32 publication-title: J Haem Pract doi: 10.17225/jhp/00178 – ident: 3068_CR8 – volume: 52 start-page: 100890 year: 2022 ident: 3068_CR35 publication-title: Blood Rev doi: 10.1016/j.blre.2021.100890 – ident: 3068_CR40 – volume: 8 start-page: 141 issue: 1 year: 2021 ident: 3068_CR50 publication-title: J Haem Pract doi: 10.2478/jhp-2021-0019 – volume: 31 start-page: 113 year: 2020 ident: 3068_CR21 publication-title: VirusDis doi: 10.1007/s13337-020-00590-2 – volume: 27 start-page: 967 year: 2021 ident: 3068_CR9 publication-title: Haemophilia doi: 10.1111/hae.14420 – volume: 9 start-page: e031763 year: 2019 ident: 3068_CR31 publication-title: BMJ Open doi: 10.1136/bmj.open-2019-031763 |
| SSID | ssj0045308 |
| Score | 2.4180439 |
| Snippet | Background
Gene therapy has the potential to offer people with haemophilia (PwH) a life free from bleeding and the burden posed by current treatment regimens.... Gene therapy has the potential to offer people with haemophilia (PwH) a life free from bleeding and the burden posed by current treatment regimens. To date,... Background Gene therapy has the potential to offer people with haemophilia (PwH) a life free from bleeding and the burden posed by current treatment regimens.... BackgroundGene therapy has the potential to offer people with haemophilia (PwH) a life free from bleeding and the burden posed by current treatment regimens.... Abstract Background Gene therapy has the potential to offer people with haemophilia (PwH) a life free from bleeding and the burden posed by current treatment... |
| SourceID | doaj pubmedcentral proquest gale pubmed crossref springer |
| SourceType | Open Website Open Access Repository Aggregation Database Index Database Enrichment Source Publisher |
| StartPage | 59 |
| SubjectTerms | Beliefs, opinions and attitudes Bureaucracy Care and treatment Clinical trials Data collection Factor IX deficiency Gene therapy Haemophilia Health care disparities Hemophilia Hemophiliacs Human Genetics Medicine Medicine & Public Health Outcomes Pharmacology/Toxicology Quality of life Shared decision making Social aspects |
| SummonAdditionalLinks | – databaseName: ProQuest Publicly Available Content dbid: PIMPY link: http://cvtisr.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwrV3db9QwDI_ghhAvfA8KAwWExAOrrp9JygvaEBOT2HTiQxpPUZqku0nQHu3dxP577DS9rUPsiZdT1bjSOXZsJ7Z_IeSVZoabPMnCMrIsBOuXh8LAxrViorDYiMkrh67_iR8eiqOjYubboztfVjnYRGeoe7RnrNsGIzw1jcYT82lSgKtmnLHs3eJXiHdIYa7VX6hxnWwg8FY0IRuz_YPZ98EyZ3kaiaFxRrBph0k7zGJmIUbOoDEj5-Qw_P-21Bdc1eUyyku5VOei9u78X-bukts-VKU7vW7dI9dsfZ_cPPDJ-AdkvqtavO-uo8uGgh5a2jdznW3T1cWWGXxNNbZHtnVH-5J1iue_dK7sz2aBRzoKnk_tW6pqan87gNAz2q3K0KHfPiTf9j58ff8x9Bc3hBq2M8tQlZpZ-LGIC6kK8JO5YUbE3IDzU1xorbm1JYSKNi4tL7kpDOLWC2ssmNso3SSTuqntY0JNXsQ25drGlc1ybcBKlKULQ-FZVCwg8SAxqT2qOV6u8UO63Y1gspeyBClLJ2WZBOTN-ptFj-lxJfUuKsKaEvG43YumPZZ-ecskrWCrzEttCpYB6wr8T1pUQlWxrYTIA_IS1Ugi4kaNJT3HatV1cv_LZ7mDMRpGZlFAXnuiqgEetPIdEjATCNI1otwaUYJJ0OPhQc2kN0mdPNeqgLxYD-OXWGZX22bV00RpCiF0QB71yr3mOxUQ8IA0AiJGaj-amPFIfTJ3gOXY4QJxZxqQ7WGFnP-vf8_8k6vZeEpuJW7tJhBLbJHJsl3ZZ-SGPl2edO1zv_b_ADqXaLY priority: 102 providerName: ProQuest – databaseName: SpringerLink dbid: RSV link: http://cvtisr.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwnV1Zb9QwEB5BQYgX7iNQkEFIPNCom8t2eGsRFUhQIQqob5ZjT9pKkFSb3Yr-e2acZGnKIcHLKorH0no8x-fY8xngmZNe-SLN42qGMqboV8Ta08K1lrpELsRUdWDXf6d2d_X-fvlhKArrxtPu45ZkiNTBrbXc7HiLjfcc85hxLs3vRbhUMNsMr9H3vozxNy-ymR7LY37bb5KCAlP_r_H4TEI6f1jy3I5pSEQ71_9vCDfg2gA8xVZvKTfhAja34Mr7YWv9Nhxu2znfXteJRSvIqlD0pVmnG2J5tgCGXwvHxY7zphP9AXTBX3PFocVv7TF_oLH0fIIvhW0Efg90n6eiW1Zx4LK9A593Xn969SYermGIHS1OFrGtnET6QWZ5tCVlvcJLrxPlKZVZpZ1zCrEi4IdJhapSvvTMQq_RIwXPWXYX1pq2wfsgfFEmmCmHSY154Tz5fFUFUEnPupYRJOPMGDdwlPNVGV9NWKtoaXoVGlKhCSo0aQQvVn2Oe4aOv0pv84SvJJldO7xo5wdmcFaTZjUtfFXlfClzGrqlbJKVtbZ1grXWRQRP2VwM82c0fEDnwC67zrzd-2i2GHExzppF8HwQqlsag7NDvQNpgim3JpLrE0lycDdtHq3SDAGmM2lJWFUqKfMInqyauScfmmuwXfYysywjQBzBvd6IV-PONMEXmo0I9MS8J4qZtjRHh4F-nOtVCEVmEWyMVv7zf_1Z8w_-TfwhXE2Do6SEFNZhbTFf4iO47E4WR938cfD4HxgAUaA priority: 102 providerName: Springer Nature |
| Title | Barriers to gene therapy, understanding the concerns people with haemophilia have: an exigency sub-study |
| URI | https://link.springer.com/article/10.1186/s13023-024-03068-2 https://www.ncbi.nlm.nih.gov/pubmed/38341591 https://www.proquest.com/docview/2925667664 https://www.proquest.com/docview/2925033435 https://pubmed.ncbi.nlm.nih.gov/PMC10859013 https://doaj.org/article/23f1847bcd964eaba46739f8af1ef885 |
| Volume | 19 |
| WOSCitedRecordID | wos001161263600001&url=https%3A%2F%2Fcvtisr.summon.serialssolutions.com%2F%23%21%2Fsearch%3Fho%3Df%26include.ft.matches%3Dt%26l%3Dnull%26q%3D |
| hasFullText | 1 |
| inHoldings | 1 |
| isFullTextHit | |
| isPrint | |
| journalDatabaseRights | – providerCode: PRVADU databaseName: BioMedCentral customDbUrl: eissn: 1750-1172 dateEnd: 99991231 omitProxy: false ssIdentifier: ssj0045308 issn: 1750-1172 databaseCode: RBZ dateStart: 20060101 isFulltext: true titleUrlDefault: https://www.biomedcentral.com/search/ providerName: BioMedCentral – providerCode: PRVAON databaseName: DOAJ Open Access Full Text customDbUrl: eissn: 1750-1172 dateEnd: 99991231 omitProxy: false ssIdentifier: ssj0045308 issn: 1750-1172 databaseCode: DOA dateStart: 20060101 isFulltext: true titleUrlDefault: https://www.doaj.org/ providerName: Directory of Open Access Journals – providerCode: PRVHPJ databaseName: ROAD: Directory of Open Access Scholarly Resources customDbUrl: eissn: 1750-1172 dateEnd: 99991231 omitProxy: false ssIdentifier: ssj0045308 issn: 1750-1172 databaseCode: M~E dateStart: 20060101 isFulltext: true titleUrlDefault: https://road.issn.org providerName: ISSN International Centre – providerCode: PRVPQU databaseName: Health & Medical Collection customDbUrl: eissn: 1750-1172 dateEnd: 99991231 omitProxy: false ssIdentifier: ssj0045308 issn: 1750-1172 databaseCode: 7X7 dateStart: 20090101 isFulltext: true titleUrlDefault: https://search.proquest.com/healthcomplete providerName: ProQuest – providerCode: PRVPQU databaseName: ProQuest Central customDbUrl: eissn: 1750-1172 dateEnd: 99991231 omitProxy: false ssIdentifier: ssj0045308 issn: 1750-1172 databaseCode: BENPR dateStart: 20090101 isFulltext: true titleUrlDefault: https://www.proquest.com/central providerName: ProQuest – providerCode: PRVPQU databaseName: Publicly Available Content Database customDbUrl: eissn: 1750-1172 dateEnd: 99991231 omitProxy: false ssIdentifier: ssj0045308 issn: 1750-1172 databaseCode: PIMPY dateStart: 20090101 isFulltext: true titleUrlDefault: http://search.proquest.com/publiccontent providerName: ProQuest – providerCode: PRVAVX databaseName: SpringerLINK Contemporary 1997-Present customDbUrl: eissn: 1750-1172 dateEnd: 99991231 omitProxy: false ssIdentifier: ssj0045308 issn: 1750-1172 databaseCode: RSV dateStart: 20061201 isFulltext: true titleUrlDefault: https://link.springer.com/search?facet-content-type=%22Journal%22 providerName: Springer Nature |
| link | http://cvtisr.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwrV1bb9MwFLZgIMQL4k5gVAYh8cCi5Wo7vK1oE5NYVXWAypPl2Cd0EqRT007s33OOk5ZmCHjhJUriEyk5N3-OfT4z9soKJ12eZGEZgQgx--WhcjhwrYQqgAoxZeXZ9T_I0UhNp8V4a6svWhPW0gO3ittP0goHIbK0rhAZmNJgZKdFpUwVQ6WUZy-NZLEeTLU5OMvTSK1LZJTYb2h6juYrs5AwMvpGrxvybP2_5-StTunqgskrs6a-Mzq6y-50KJIftG9_j12D-j67ddLNkz9gs6FZ0FZ0DV_OOboI8LbO6nKPr7arWeg2t1S5uKgb3q4m5_Rrls8MfJ-f098Wg-cX8JabmsMPz915yZtVGXpi2ofs09Hhx3fvw25PhdDiSGMZmtIK1KIFomw0BXZhuRNOxdJhv2SkstZKgBJRHMQlyFK6whGlvAIHmAmj9BHbqec1PGHc5UUMqbQQV5Dl1mEAl6VHiHiuKhGweK1ibTvCcdr34pv2Aw8ldGsWjWbR3iw6CdibzTPnLd3GX6WHZLmNJFFl-xvoQLpzIP0vBwrYS7K7JjKMmlbbfDWrptHHpxN9QPCJQFMUsNedUDXHb7CmK15ATRB_Vk9ytyeJ0Wr7zWv30l22aHRSIPAUUogsYC82zfQkrYCrYb5qZaI0RXQbsMetN26-O1WIRdAaAVM9P-0ppt9Sn808lzgVnyAkTAO2t3bpX-_1Z80__R-af8ZuJz4kEwQDu2xnuVjBc3bTXizPmsWAXZdT6Y9qwG4MD0fjycAHOV6Nj0_GX_Bqcvr5J0h5VHI |
| linkProvider | Directory of Open Access Journals |
| linkToHtml | http://cvtisr.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMw1V1Lb9NAEB6VFAEX3g9DgQWBOFCrjp9rJIRaoGrUJIpokcppsXfXTSWwQ5wU8qf4jcys7bQuorceuESWdxx51zPfzOzOA-CFDFWkAte3U0eHNqJfYHOFjmsW8lhTImaUmer6_Wg45AcH8WgFfje5MBRW2WCiAWpVSNoj33BjVM5hFIb-u8kPm7pG0elq00KjYotdvfiJLlv5tvcBv-9L193-uP9-x667CtgSbe2ZnaQy1PijqWhhEiOIBypUvBspROYk4lLKSOsU7RjdTXWURipWVFSda6URCxwP__cSrPrI7E4HVke9wehLg_1-4Dm8Sc3h4UZJx4J0TurbZJsjT7bUn-kS8LcuOKUMzwZqnjmtNUpw-8b_tnw34XptbrPNSj5uwYrOb8OVQR1QcAfGW8mUevaVbFYwlCXNqoS0xTqbn077odtMUornNC9ZFXbPaA-bjRP9vZjQtlSC18f6DUtypn-ZIqcLVs5T21TwvQufL2Se96CTF7l-AEwFcVd7kdTdTPuBVIh0aWpMabzmWWhBt-EJIevK7NQg5JswHhoPRcVHAvlIGD4SrgWvl89Mqrok51JvEastKammuLlRTA9FDVHC9TJ096NUqjj0ceoJ6lAvzniSdXXGeWDBc2JUQVVDcgpLOkzmZSl6e5_EJtmZZF06FryqibIC5yCTOssDV4IKjbUo11qUCGuyPdwwsqhhtRQnXGzBs-UwPUmhgrku5hWN43noBlhwvxKf5bw9jkYbfg0LeEuwWgvTHsmPxqboOmXpoO3sWbDeyODJe_175R-eP42ncHVnf9AX_d5w9xFccw1SuGgbrUFnNp3rx3BZHs-OyumTGmkYfL1o6fwDHS29-w |
| linkToPdf | http://cvtisr.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwpV1Lb9QwEB5BQRUX3o9AAYOQONCom5ftcGuBioqyqlpAvVmO7XQrQbLa7Fb03zPjJMumPCTEZRXFY2k9Hs98jmc-A7ww3AqbxWlYjBwP0ftlobS4cS25zB0VYorSs-vvi_FYHh_nBytV_D7bvT-SbGsaiKWpmm9Nbdkuccm3Gjpuo_PHNCTMi3N9Ga6kuJOhpK7Doy-9L06zZCT7Upnf9huEI8_a_6tvXglOFxMnL5ye-qC0e-P_h3MTrneAlG23FnQLLrnqNqx_7I7c78BkR8_oVruGzWuG1uZYW7J1vskWq4Ux9JoZKoKcVQ1rE9MZfeVlE-2-1VP6cKPx-cy9Zrpi7runAT1nzaIIPcftXfi8--7Tm_dhdz1DaHDTMg91YbjDH0fsjzrHaJhZbmUkLIY4LaQxRjhXICB0UeFEIWxuiZ1eOuvQqY6Se7BW1ZV7AMxmeeQSYVxUujQzFn1BUXiwic-y5AFE_Swp03GX0xUaX5Xfw0iuWhUqVKHyKlRxAK-WfaYtc8dfpXdo8peSxLrtX9SzE9UtYhUnJW6IRWFszlMcusYok-Sl1GXkSimzAJ6T6Sji1agocedEL5pG7R0dqm1CYoS_RgG87ITKGsdgdFcHgZogKq6B5MZAEhe-GTb3Fqo6x9OoOEcMywXnaQDPls3Uk5LpKlcvWplRkiBQDuB-a9DLcScSYQ3ORgByYOoDxQxbqtOJpyWnOhZEl0kAm73F__xff9b8w38TfwrrB2931f7e-MMjuBb7NRMjmNiAtfls4R7DVXM2P21mT7wj-AEx5V1o |
| openUrl | ctx_ver=Z39.88-2004&ctx_enc=info%3Aofi%2Fenc%3AUTF-8&rfr_id=info%3Asid%2Fsummon.serialssolutions.com&rft_val_fmt=info%3Aofi%2Ffmt%3Akev%3Amtx%3Ajournal&rft.genre=article&rft.atitle=Barriers+to+gene+therapy%2C+understanding+the+concerns+people+with+haemophilia+have%3A+an+exigency+sub-study&rft.jtitle=Orphanet+journal+of+rare+diseases&rft.au=Fletcher%2C+Simon&rft.au=Jenner%2C+Kathryn&rft.au=Holland%2C+Michael&rft.au=Khair%2C+Kate&rft.date=2024-02-10&rft.issn=1750-1172&rft.eissn=1750-1172&rft.volume=19&rft.issue=1&rft_id=info:doi/10.1186%2Fs13023-024-03068-2&rft.externalDBID=n%2Fa&rft.externalDocID=10_1186_s13023_024_03068_2 |
| thumbnail_l | http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/lc.gif&issn=1750-1172&client=summon |
| thumbnail_m | http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/mc.gif&issn=1750-1172&client=summon |
| thumbnail_s | http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/sc.gif&issn=1750-1172&client=summon |