Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
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Reversible immortalisation enables genetic correction of human muscle progenitors and engineering of next-generation human artificial chromosomes for Duchenne muscular dystrophy.A mouse anti-myostatin antibody increases muscle mass and improves muscle strength and contractility in the mdx mouse model of Duchenne muscular dystrophy and its humanized equivalent, domagrozumab (PF-06252616), increases muscle volume in cynomolguPharmacological inhibition of REV-ERB stimulates differentiation, inhibits turnover and reduces fibrosis in dystrophic muscle.Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management.Treating pediatric neuromuscular disorders: The future is now.The effect of PTC124 on choroideremia fibroblasts and iPSC-derived RPE raises considerations for therapy.At the Crossroads of Clinical and Preclinical Research for Muscular Dystrophy-Are We Closer to Effective Treatment for Patients?A G542X cystic fibrosis mouse model for examining nonsense mutation directed therapies.Advances in therapeutic use of a drug-stimulated translational readthrough of premature termination codons.Ambulatory function in spinal muscular atrophy: Age-related patterns of progression.Off-Label Use of Ataluren in Four Non-ambulatory Patients With Nonsense Mutation Duchenne Muscular Dystrophy: Effects on Cardiac and Pulmonary Function and Muscle StrengthThe Pathogenesis and Therapies of Striated Muscle LaminopathiesDystrophin Cardiomyopathies: Clinical Management, Molecular Pathogenesis and Evolution towards Precision MedicineGene and Cell Therapy for Muscular Dystrophies: Are We Getting There?
P2860
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P2860
Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
description
2017 nî lūn-bûn
@nan
2017年の論文
@ja
2017年学术文章
@wuu
2017年学术文章
@zh-cn
2017年学术文章
@zh-hans
2017年学术文章
@zh-my
2017年学术文章
@zh-sg
2017年學術文章
@yue
2017年學術文章
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2017年學術文章
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name
Ataluren in patients with nons ...... ebo-controlled, phase 3 trial.
@en
type
label
Ataluren in patients with nons ...... ebo-controlled, phase 3 trial.
@en
prefLabel
Ataluren in patients with nons ...... ebo-controlled, phase 3 trial.
@en
P2093
P50
P921
P1433
P1476
Ataluren in patients with nons ...... ebo-controlled, phase 3 trial.
@en
P2093
ACT DMD Study Group
Andrés Nascimento Osorio
Anna Kaminska
Brenda Wong
Clinical Evaluator Training Group
Craig Campbell
Craig M McDonald
Eugenio Mercuri
Francesco Muntoni
Gary Elfring
P356
10.1016/S0140-6736(17)31611-2
P407
P577
2017-07-17T00:00:00Z