Short-term safety results from compassionate use of risdiplam in patients with spinal muscular atrophy in Germany

BACKGROUND: The oral, selective SMN2-splicing modifier risdiplam obtained European approval in March 2021 for the treatment of patients ≥ 2 months old with a clinical diagnosis of 5q-associated spinal muscular atrophy (SMA) 1/2/3 or with 1–4 SMN2 gene copies. For the preceding 12 months, this compas...

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Detalles Bibliográficos
Autores principales: Hahn, Andreas, Günther, René, Ludolph, Albert, Schwartz, Oliver, Trollmann, Regina, Weydt, Patrick, Weiler, Markus, Neuland, Kathrin, Schwaderer, Martin Sebastian, Hagenacker, Tim
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9295446/
https://www.ncbi.nlm.nih.gov/pubmed/35854272
http://dx.doi.org/10.1186/s13023-022-02420-8

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