Results 241 to 250 of about 104,787 (267)

Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial

Nature Medicine, 2022
Kevin A Strauss, Michelle A Farrar Mbbs, Francesco Muntoni   +2 more
exaly  

Therapeutic approaches for Duchenne muscular dystrophy

Nature Reviews Drug Discovery, 2023
Thomas C Roberts, Kay E Davies
exaly  

Spinal muscular atrophy

Nature Reviews Disease Primers, 2022
Francesco Muntoni, Richard S Finkel
exaly  

Muscular dystrophies

Disease-a-Month, 1972
openaire   +2 more sources

MUSCULAR DYSTROPHIES

Continuum, 2003
openaire   +1 more source

Muscular dystrophies

The Indian Journal of Pediatrics, 1990
openaire   +2 more sources

Muscular Dystrophies

Pediatric Annals, 2005
openaire   +2 more sources

Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy

Nature Medicine, 2020
Alessandra Moretti, Daniel Sinnecker, Shicheng Sun   +2 more
exaly  

Muscular Dystrophies

1984
Michael Swash, Martin S. Schwartz
openaire   +1 more source

Congenital Muscular Dystrophy

Pediatric Annals, 2005
Eugenio, Mercuri, Cheryl, Longman
openaire   +2 more sources