Results 271 to 280 of about 71,159 (294)

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  

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  

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  

Carnitine in duchenne muscular dystrophy

Clinica Chimica Acta, 1979
Mawatari Shiro, Kobayashi Takuro, Shinnoh Nobue   +2 more
openaire   +3 more sources

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  

Detection of collagens by multispectral optoacoustic tomography as an imaging biomarker for Duchenne muscular dystrophy

Nature Medicine, 2019
Adrian P Regensburger, Jörg Jüngert, Teresa Gerhalter   +2 more
exaly  

Steroids in Duchenne muscular dystrophy

Neuromuscular Disorders, 2013
Valeria Ricotti, Deborah Ridout, Francesco Muntoni   +2 more
openaire   +3 more sources

Defective membrane repair in dysferlin-deficient muscular dystrophy

Nature, 2003
Steven S Vogel, Chien-Chang Chen, Kevin P Campbell   +2 more
exaly