Found: 11
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Health-Related Quality of Life and Emotional Distress Among Mothers of Sons With Muscular Dystrophy as Compared to Sex- and Age Group–Matched Controls.
- Published in:
- Journal of Child Neurology, 2021, v. 36, n. 3, p. 177, doi. 10.1177/0883073820962927
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- Article
Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy.
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- Pediatric Pulmonology, 2019, v. 54, n. 2, p. 179, doi. 10.1002/ppul.24203
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- Article
Clinical Follow-Up for Duchenne Muscular Dystrophy Newborn Screening: A Proposal.
- Published in:
- 2016
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- journal article
Milder phenotype of congenital muscular dystrophy in a novel POMT1 mutation.
- Published in:
- Muscle & Nerve, 2012, v. 45, n. 5, p. 752, doi. 10.1002/mus.23274
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- Article
A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy.
- Published in:
- Molecular Therapy, 2015, v. 23, n. 1, p. 192, doi. 10.1038/mt.2014.200
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- Article
Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy.
- Published in:
- JAMA Neurology, 2021, v. 78, n. 7, p. 834, doi. 10.1001/jamaneurol.2021.1272
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- Article
Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial.
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- JAMA Neurology, 2020, v. 77, n. 9, p. 1122, doi. 10.1001/jamaneurol.2020.1484
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- Article
Neurological Complications of PCR-Proven M. pneumoniae Infections in Children: Prodromal Illness Duration May Reflect Pathogenetic Mechanism.
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- Clinical Infectious Diseases, 2015, v. 61, n. 7, p. 1092, doi. 10.1093/cid/civ473
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- Article
Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy.
- Published in:
- 2016
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- Publication type:
- journal article
ACTIVE (Ability Captured Through Interactive Video Evaluation) workspace volume video game to quantify meaningful change in spinal muscular atrophy.
- Published in:
- 2020
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- Publication type:
- journal article
A slowly progressive form of limb-girdle muscular dystrophy type 2C associated with founder mutation in the SGCG gene in Puerto Rican Hispanics.
- Published in:
- Molecular Genetics & Genomic Medicine, 2015, v. 3, n. 2, p. 92, doi. 10.1002/mgg3.125
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- Publication type:
- Article