Results 51 to 60 of about 136 (71)

Relatório Final de Estágio [PDF]

Inês Cunha Moreira
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Duchenne muscular dystrophy

Nature Reviews Disease Primers, 2021
Dongsheng Duan, Nathalie Goemans, Shin’ichi Takeda   +2 more
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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
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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
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Spinal muscular atrophy

Nature Reviews Disease Primers, 2022
Francesco Muntoni, Richard S Finkel
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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
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Induced pluripotent stem cells from a spinal muscular atrophy patient

Nature, 2008
Allison D Ebert, Christian L Lorson, Clive N Svendsen   +2 more
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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
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Defective membrane repair in dysferlin-deficient muscular dystrophy

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