Amifampridine safety and efficacy in spinal muscular atrophy ambulatory patients: a randomized, placebo-controlled, crossover phase 2 trial

BACKGROUND: Spinal muscular atrophy (SMA) is an autosomal recessive disease where a deficient amount of SMN protein leads to progressive lower motor neuron degeneration. SMN-enhancing therapies are now available. Yet, fatigue and signs of impaired neuromuscular junction (NMJ) transmission could cont...

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Detalles Bibliográficos
Autores principales: Bonanno, Silvia, Giossi, Riccardo, Zanin, Riccardo, Porcelli, Valentina, Iannacone, Claudio, Baranello, Giovanni, Ingenito, Gary, Iyadurai, Stanley, Stevic, Zorica, Peric, Stojan, Maggi, Lorenzo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Berlin Heidelberg 2022
Materias:
Original Communication
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9243784/
https://www.ncbi.nlm.nih.gov/pubmed/35763114
http://dx.doi.org/10.1007/s00415-022-11231-7

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9243784/
https://www.ncbi.nlm.nih.gov/pubmed/35763114
http://dx.doi.org/10.1007/s00415-022-11231-7

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