Results 161 to 170 of about 2,169 (197)

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
openaire   +2 more sources

Risdiplam bei SMA zugelassen

NeuroTransmitter, 2021
openaire   +1 more source

Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls

New England Journal of Medicine, 2021
Riccardo Masson, Maria Mazurkiewicz-Bełdzińska, Giovanni Baranello   +2 more
exaly  

Zulassungserweiterung für Risdiplam

DNP – Die Neurologie & Psychiatrie, 2023
openaire   +1 more source

Orale Therapie der SMA mit Spleißmodulator Risdiplam

DNP - Der Neurologe & Psychiater, 2022
Svenja Brakemeier, Tim Hagenacker
openaire   +1 more source

Risdiplam von Geburt an

InFo Neurologie + Psychiatrie, 2023
openaire   +1 more source

Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial

Lancet Neurology, The, 2022
Eugenio Mercuri, Elena Mazzone, Andrés Nascimento   +2 more
exaly  

Nusinersen/onasemnogene-abeparvovec/risdiplam

Reactions Weekly, 2022
openaire   +1 more source

Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial

Lancet Neurology, The, 2022
Riccardo Masson, Laurent J Servais, Giovanni Baranello   +2 more
exaly  

P.113 Safety update: Risdiplam clinical trial development program

Neuromuscular Disorders, 2022
Chiriboga, Claudia A., Servais, Laurent, Baranello, Giovanni, Darras, Basil T., Day, James M. D., Deconinck, Nicolas, Farrar, M., Finkel, R., Bertini, Enrico, Kirschner, Janbernd, Masson, Riccardo, Mazurkiewicz-Bełdzińska, M., Vlodavets, D., Bader‐Weder, Silvia, Gorni, Ksenija, Jaber, B., McIver, Tammy, Papp, G., Scalco, Renata S., Mercuri, Eugenio   +19 more
openaire   +2 more sources