Results 141 to 150 of about 110,882 (159)

Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial

Nature Medicine, 2022
Kevin A Strauss, Michelle A Farrar Mbbs
exaly  

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  

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  

Induced pluripotent stem cells from a spinal muscular atrophy patient

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

Defective membrane repair in dysferlin-deficient muscular dystrophy

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