Results 281 to 290 of about 92,711 (302)

Delay in Diagnosis of Spinal Muscular Atrophy: A Systematic Literature Review

Pediatric Neurology, 2015
Chia-Wei Lin
exaly  

SCAPULOPERONEAL MUSCULAR ATROPHY

Brain, 1965
openaire   +2 more sources

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, Jiří Vajsar   +2 more
exaly  

Phenotypic and molecular insights into spinal muscular atrophy due to mutations in BICD2

Brain, 2015
Alexander M Rossor, Emily C Oates, Sinéad M Murphy   +2 more
exaly  

Distal infantile spinal muscular atrophy associated with paralysis of the diaphragm: A variant of infantile spinal muscular atrophy

American Journal of Medical Genetics Part A, 1989
Enrico Bertini, G Ferriere
exaly  

Onasemnogene Abeparvovec-xioi: Gene Therapy for Spinal Muscular Atrophy

Annals of Pharmacotherapy, 2020
Debra L Stevens, Cheri K Walker
exaly  

Spinal muscular atrophy: going beyond the motor neuron

Trends in Molecular Medicine, 2013
Thomas H Gillingwater
exaly  

Spinal muscular atrophy and pregnancy

BJOG: An International Journal of Obstetrics & Gynaecology, 1992
R D, Wilson, K P, Williams
openaire   +2 more sources

Nusinersen: A Treatment for Spinal Muscular Atrophy

Annals of Pharmacotherapy, 2019
Debra L Stevens, Cheri K Walker
exaly  

The cost-effectiveness of prenatal screening for spinal muscular atrophy

American Journal of Obstetrics and Gynecology, 2010
Sarah E Little, Vanitha Janakiraman, Anjali Kaimal   +2 more
exaly