Results 261 to 270 of about 605,611 (288)

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
Claudia Dosi, Valeria Ada María Sansone, Martina Ricci   +2 more
exaly  

Spinal Muscular Atrophy: Mutations, Testing, and Clinical Relevance

The Application of Clinical Genetics, 2021
Thomas W Prior
exaly  

Spinal Muscular Atrophy

Pediatric Annals, 1989
openaire   +2 more sources

Spinal Muscular Atrophy Treatment in Patients Identified by Newborn Screening—A Systematic Review

Genes, 2023
Laurent Servais, Charlotte Mouraux, Tamara Dangouloff   +2 more
exaly  

New Treatments in Spinal Muscular Atrophy: Positive Results and New Challenges

Journal of Clinical Medicine, 2020
Sonia Messina
exaly  

Experience and Perspectives in the US on the Evolving Treatment Landscape in Spinal Muscular Atrophy

International Journal of General Medicine, 2022
Perry Shieh
exaly  

Epidemiological investigation of spinal muscular atrophy in Japan

Brain and Development, 2022
Tamaki Kato, Mari Matsuo, Mari Urano
exaly  

The Spinal Muscular Atrophies

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

Spinal Muscular Atrophies

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

New and Developing Therapies in Spinal Muscular Atrophy: From Genotype to Phenotype to Treatment and Where Do We Stand?

International Journal of Molecular Sciences, 2020
Tai-Heng Chen
exaly