Results 191 to 200 of about 888,799 (202)

Onasemnogene Abeparvovec: Post-infusion Efficacy and Safety in Patients With Spinal Muscular Atrophy (SMA)-A Fondazione Policlinico Gemelli IRCCS Experience. [PDF]

Hosp Pharm
Favia M, Tarantino D, Cerbo LD, Sabia A, Campopiano R, Pani M.   +5 more
europepmc   +1 more source

Routine Cerebrospinal Fluid (CSF) Parameters in Patients With Spinal Muscular Atrophy (SMA) Treated With Nusinersen. [PDF]

Front Neurol, 2019
Wurster CD, Koch JC, Cordts I, Dreyhaupt J, Otto M, Uzelac Z, Witzel S, Winter B, Kocak T, Schocke M, Weydt P, Wollinsky K, Ludolph AC, Deschauer M, Lingor P, Tumani H, Hermann A, Günther R.   +17 more
europepmc   +1 more source

An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials. [PDF]

CNS Drugs, 2019
Darras BT, Farrar MA, Mercuri E, Finkel RS, Foster R, Hughes SG, Bhan I, Farwell W, Gheuens S.   +8 more
europepmc   +1 more source

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Risdiplam: an investigational survival motor neuron 2 (SMN2) splicing modifier for spinal muscular atrophy (SMA)

Expert Opinion on Investigational Drugs, 2022
Laurent Servais, Sithara Ramdas, Gemma Fisher   +2 more
exaly  

Disease Modifying Therapies for the Management of Children with Spinal Muscular Atrophy (5q SMA): An Update on the Emerging Evidence

Drug Design, Development and Therapy, 2022
Thomas Sejersen, Helgi Thor Hjartarson
exaly  

Use of orthoses and orthopaedic technical devices in proximal spinal muscular atrophy. Results of survey in 194 SMA patients

Disability and Rehabilitation: Assistive Technology, 2011
Jürgen Forst
exaly  

Spinal muscular atrophy: going beyond the motor neuron

Trends in Molecular Medicine, 2013
Thomas H. Gillingwater
exaly  

Nusinersen for the treatment of spinal muscular atrophy

Expert Review of Neurotherapeutics, 2017
Claudia Chiriboga
exaly  

Evaluating Benefit-risk Decision-making in Spinal Muscular Atrophy: A First-ever Study to Assess Risk Tolerance in the SMA Patient Community

Clinical Therapeutics, 2019
Rosángel Cruz, Lisa Belter, Jill Jarecki   +2 more
exaly