Results 151 to 160 of about 2,038 (184)

182 Pooled safety data from the risdiplam clinical development programme

Journal of Neurology, Neurosurgery & Psychiatry, 2022
Risdiplam (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, Laurent Servais, Claudia Chiriboga, Basil Darras, Silvia Bader-Weder, Ksenija Gorni, Birgit Jaber, Tammy McIver, Renata Scalco, Eugenio Mercuri   +9 more
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Risdiplam in Presymptomatic Spinal Muscular Atrophy

New England Journal of Medicine
Background 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 ...
Finkel, RS, Servais, L, Vlodavets, D, Zanoteli, E, Mazurkiewicz-Bełdzińska, M, Jong, Y-J, Navas-Nazario, A, Al-Muhaizea, M, Araujo, APQC, Nelson, L, Wang, Y, Jaber, B, Gorni, K, Kletzl, H, Palfreeman, L, Rabbia, M, Summers, D, Gaki, E, Wagner, KR, Fontoura, P, Farrar, MA, Bertini, E   +21 more
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Risdiplam for the treatment of spinal muscular atrophy

S.S. Korsakov Journal of Neurology and Psychiatry
Spinal 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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Risdiplam

Reactions Weekly, 2023
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Risdiplam

Reactions Weekly
  +5 more sources

Nusinersen/risdiplam

Reactions Weekly, 2023
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Clinical Application of Risdiplam in 5q Spinal Muscular Atrophy: A Narrative Review

British Journal of Hospital Medicine
5q spinal muscular atrophy (SMA) is caused by mutations in the survival motor neuron (SMN) gene located on chromosome 5, leading to insufficient SMN protein levels. Risdiplam is an RNA splicing modifier that modifies pre-mRNA splicing of the SMN2 gene, thereby promoting the production of functional survival motor neuron protein (SMN-fl).
Tong, Zhao, Yang, Xue, Miao-Shui, Bai, Han-Yu, Dong, Fei-Yong, Jia   +4 more
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Risdiplam bei SMA zugelassen

NeuroTransmitter, 2021
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Zulassungserweiterung für Risdiplam

DNP – Die Neurologie & Psychiatrie, 2023
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Orale Therapie der SMA mit Spleißmodulator Risdiplam

DNP - Der Neurologe & Psychiater, 2022
Svenja Brakemeier, Tim Hagenacker
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