Výsledky vyhledávání - "Valentina Porcelli"

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

Autor: Silvia Bonanno, Riccardo Giossi, Riccardo Zanin, Valentina Porcelli, Claudio Iannacone, Giovanni Baranello, Gary Ingenito, Stanley Iyadurai, Zorica Stevic, Stojan Peric, Lorenzo Maggi

Publikováno v: Journal of neurology. 269(11)

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

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::37cabb275b2d4cfc0fa15a9ae2bb8b3a
https://pubmed.ncbi.nlm.nih.gov/35763114

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