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...

Full description

Saved in:

Bibliographic Details
Published in:	Annals of Clinical and Translational Neurology Vol. 10; no. 11; pp. 2155 - 2160
Main Authors:	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
Format:	Journal Article
Language:	English
Published:	Bognor Regis Wiley 01.11.2023 John Wiley & Sons, Inc John Wiley and Sons Inc
Subjects:	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
Online Access:	Get full text
Tags:	Add Tag No Tags, Be the first to tag this record!

Be the first to leave a comment!