Combination disease‐modifying treatment in spinal muscular atrophy: A proposed classification

We sought to devise a rational, systematic approach for defining/grouping survival motor neuron ‐targeted disease‐modifying treatment (DMT) scenarios. The proposed classification is primarily based on a two‐part differentiation: initial DMT, and persistence/discontinuation of subsequent DMT(s). Trea...

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Podrobná bibliografie
Vydáno v:	Annals of Clinical and Translational Neurology Ročník 10; číslo 11; s. 2155 - 2160
Hlavní autoři:	Proud, Crystal M., Mercuri, Eugenio, Finkel, Richard S., Kirschner, Janbernd, De Vivo, Darryl C., Muntoni, Francesco, Saito, Kayoko, Tizzano, Eduardo F., Desguerre, Isabelle, Quijano‐Roy, Susana, Benguerba, Kamal, Raju, Dheeraj, Faulkner, Eric, Servais, Laurent
Médium:	Journal Article
Jazyk:	angličtina
Vydáno:	Bognor Regis Wiley 01.11.2023 John Wiley & Sons, Inc John Wiley and Sons Inc
Témata:	Age Atrophy Atròfia muscular espinal - Tractament Brief Communications Classification Clinical outcomes enfermedades del sistema nervioso::enfermedades del sistema nervioso central::enfermedades de la médula espinal::atrofia muscular espinal [ENFERMEDADES] Gene therapy Human health sciences Humans Investigative Techniques::Epidemiologic Methods::Data Collection::Registries [ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT] Medical screening Muscular Atrophy, Spinal Muscular Atrophy, Spinal/drug therapy Nervous System Diseases::Central Nervous System Diseases::Spinal Cord Diseases::Muscular Atrophy, Spinal [DISEASES] Neurology (clinical) Neurology. Diseases of the nervous system Neuroscience (all) Neurosciences. Biological psychiatry. Neuropsychiatry Other subheadings::Other subheadings::/drug therapy [Other subheadings] Otros calificadores::Otros calificadores::/farmacoterapia [Otros calificadores] Patients Pediatrics Pédiatrie RC321-571 RC346-429 Registres mèdics Registries Sciences de la santé humaine técnicas de investigación::métodos epidemiológicos::recopilación de datos::registros [TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS]
ISSN:	2328-9503, 2328-9503
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Popis
Shrnutí:	We sought to devise a rational, systematic approach for defining/grouping survival motor neuron ‐targeted disease‐modifying treatment (DMT) scenarios. The proposed classification is primarily based on a two‐part differentiation: initial DMT, and persistence/discontinuation of subsequent DMT(s). Treatment categories were identified: monotherapy add‐on, transient add‐on, combination with onasemnogene abeparvovec, bridging to onasemnogene abeparvovec, and switching to onasemnogene abeparvovec. We validated this approach by applying the classification to the 443 patients currently in the RESTORE registry and explored the demographics of these different groups of patients. This work forms the basis to explore the safety and efficacy profile of the different combinations of DMT in SMA.
Bibliografie:	ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 14 content type line 23 scopus-id:2-s2.0-85169689385
ISSN:	2328-9503 2328-9503
DOI:	10.1002/acn3.51889