Results 271 to 280 of about 148,459 (298)

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  

Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study

Lancet, The, 2016
Richard S Finkel, Claudia A Chiriboga, John W Day   +2 more
exaly  

Universal newborn screening for spinal muscular atrophy in Ukraine

Lancet, The, 2023
exaly  

SCAPULOPERONEAL MUSCULAR ATROPHY

Brain, 1965
openaire   +2 more sources

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

New England Journal of Medicine, 2017
Richard S Finkel, Eugenio Mercuri, Anne M Connolly   +2 more
exaly  

Spinal muscular atrophy

International Journal of Obstetric Anesthesia, 2003
openaire   +2 more sources

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

New England Journal of Medicine, 2017
Richard Shell, Lindsay N Alfano, Shibi Likhite   +2 more
exaly  

Spinal Muscular Atrophies

2005
Klaus Zerres, Sabine Rudnik-Schöneborn
openaire   +1 more source

Induced pluripotent stem cells from a spinal muscular atrophy patient

Nature, 2008
Christian L Lorson, Clive N Svendsen
exaly  

Muscular atrophy

The Indian Journal of Pediatrics, 1939
openaire   +1 more source