Treatment expectations and patient-reported outcomes of nusinersen therapy in adult spinal muscular atrophy

BACKGROUND: The antisense-oligonucleotide (ASO) nusinersen has recently been approved as the first genetically modifying therapy for 5q-associated spinal muscular atrophy (SMA) based on randomized sham-controlled trials in infants and children. The efficacy in adults with long disease history and ad...

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Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:J Neurol
Egile Nagusiak: Osmanovic, Alma, Ranxha, Gresa, Kumpe, Mareike, Müschen, Lars, Binz, Camilla, Wiehler, Flavia, Paracka, Lejla, Körner, Sonja, Kollewe, Katja, Petri, Susanne, Schreiber-Katz, Olivia
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: Springer Berlin Heidelberg 2020
Gaiak:
Original Communication
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC7359174/
https://ncbi.nlm.nih.gov/pubmed/32361837
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s00415-020-09847-8
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