Results 151 to 160 of about 2,169 (197)
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Drugs, 2020
Risdiplam (Evrysdi™) is an orally administered, survival motor neuron 2 (SMN2)-directed RNA splicing modifier being developed by Roche, PTC Therapeutics Inc and the SMA Foundation for the treatment of the spinal muscular atrophy. The small molecule is designed to treat spinal muscular atrophy caused by mutations in chromosome 5q leading to SMN protein ...
exaly +3 more sources
Risdiplam (Evrysdi™) is an orally administered, survival motor neuron 2 (SMN2)-directed RNA splicing modifier being developed by Roche, PTC Therapeutics Inc and the SMA Foundation for the treatment of the spinal muscular atrophy. The small molecule is designed to treat spinal muscular atrophy caused by mutations in chromosome 5q leading to SMN protein ...
exaly +3 more sources
Risdiplam: A Review in Spinal Muscular Atrophy
CNS Drugs, 2022Risdiplam (Evrysdi®) is the first oral drug developed to treat spinal muscular atrophy (SMA) and is approved in multiple countries worldwide. It is approved for the treatment of SMA in patients aged ≥ 2 months in the USA and the EU, with this approval further specified in the EU for the treatment of 5q-autosomal recessive SMA with a clinical diagnosis ...
exaly +3 more sources
Risdiplam in Presymptomatic Spinal Muscular Atrophy
New England Journal of MedicineBackground Risdiplam, an oral pre–messenger RNA splicing modifier, is an efficacious treatment for persons with symptomatic spinal muscular atrophy (SMA). The safety and efficacy of risdiplam in presymptomatic disease are unclear. Methods We conducted an open-label study of daily oral risdiplam (with the dose adjusted to 0.2 mg per kilogram of body ...
Richard S Finkel +2 more
exaly +2 more sources
Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA) [PDF]
Journal of Neurology, 2023 Risdiplam is an oral, survival of motor neuron 2 (SMN2) pre-mRNA splicing modifier approved for the treatment of spinal muscular atrophy (SMA). SUNFISH (NCT02908685) Part 2, a Phase 3, randomized, double-blind, placebo-controlled study, investigated the ...
Maryam Oskoui +2 more
exaly +6 more sources
Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy
New England Journal of MedicineRichard S, Finkel +7 more
exaly +3 more sources
182 Pooled safety data from the risdiplam clinical development programme
Journal of Neurology, Neurosurgery & Psychiatry, 2022Risdiplam (EVRYSDI®) is a centrally and peripherally distributed, oral survival of motor neuron 2 (SMN2) premRNA splicing modifier approved by the EMA and MHRA for the treatment of patients aged ≥2 months, with a clinical diagnosis of Type 1, 2 or 3 spinal muscular atrophy (SMA) or 1–4 copies of SMN2.Safety data were pooled from three studies within ...
Giovanni Baranello +9 more
openaire +1 more source
Risdiplam for the treatment of spinal muscular atrophy
S.S. Korsakov Journal of Neurology and PsychiatrySpinal muscular atrophy (SMA) is a devastating disease that is the leading genetic cause of death in infants and young children. It includes a broad spectrum of phenotypes that are classified into clinical groups based on the age of onset and maximum motor function achieved.
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