Results 231 to 240 of about 45,438 (256)

Counteracting chromatin effects of a splicing-correcting antisense oligonucleotide improves its therapeutic efficacy in spinal muscular atrophy

Cell, 2022
Luciano E Marasco, Gwendal Dujardin, Rui Sousa-Luís   +2 more
exaly  

Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapies

EClinicalMedicine, 2023
Anna Capasso, Antonio Varone, Caterina Ágosto   +2 more
exaly  

Spinal Muscular Atrophy

Pediatric Annals, 1989
openaire   +2 more sources

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
exaly  

New treatments in spinal muscular atrophy: an overview of currently available data

Expert Opinion on Pharmacotherapy, 2020
Sithara Ramdas, Laurent Servais
exaly  

Spinal muscular atrophy and pregnancy

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

<p>Clinical Evidence Supporting Early Treatment Of Patients With Spinal Muscular Atrophy: Current Perspectives</p>

Therapeutics and Clinical Risk Management, 2019
Tamara Dangouloff, Laurent Servais
exaly  

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

Pediatric Neurology, 2015
Chia-Wei Lin
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, Jiří Vajsar   +2 more
exaly  

The Spinal Muscular Atrophies

Developmental Medicine & Child Neurology, 1991
openaire   +2 more sources