Results 331 to 340 of about 182,997 (357)

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, Francesco Muntoni
exaly  

Muscular Dystrophies

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

Therapeutic approaches for Duchenne muscular dystrophy

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

Muscular dystrophies

The Indian Journal of Pediatrics, 1990
openaire   +3 more sources

Spinal muscular atrophy

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

Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy

Nature Medicine, 2019
Christopher E Nelson, Yaoying Wu, Matthew A Waller   +2 more
exaly  

Induced pluripotent stem cells from a spinal muscular atrophy patient

Nature, 2008
Allison D Ebert, Christian L Lorson, Clive N Svendsen   +2 more
exaly  

Therapeutic developments for Duchenne muscular dystrophy

Nature Reviews Neurology, 2019
Ingrid E C Verhaart, Annemieke Aartsma-Rus   +1 more
exaly  

Muscular dystrophies

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

MUSCULAR DYSTROPHIES

The Lancet, 1974
Stanley H. Appel, Allen D. Roses
openaire   +3 more sources