Results 201 to 210 of about 47,298 (216)
Some of the next articles are maybe not open access.

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   +2 more
exaly  

Base editing rescue of spinal muscular atrophy in cells and in mice

Science, 2023
Mandana Arbab   +2 more
exaly  

Pharmacological strategies for muscular dystrophy

Nature Reviews Drug Discovery, 2003
Tejvir S Khurana, Kay E Davies
exaly  

Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study

Lancet, The, 2018
Craig M Mcdonald   +2 more
exaly  

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

Lancet, The, 2017
Craig M Mcdonald   +2 more
exaly  

Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial

Lancet, The, 2015
Maria Grazia D'Angelo   +2 more
exaly  

Induced pluripotent stem cells from a spinal muscular atrophy patient

Nature, 2008
Allison D Ebert   +2 more
exaly  

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

Nature Medicine, 2019
Adrian P Regensburger   +2 more
exaly  

Therapeutic developments for Duchenne muscular dystrophy

Nature Reviews Neurology, 2019
Ingrid E C Verhaart   +1 more
exaly  

Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management

Lancet Neurology, The, 2018
David J Birnkrant   +2 more
exaly  
músculo liso vascular
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