F1000Res. 2016 Aug 18;5. pii: F1000 Faculty Rev-2030. doi: 10.12688/f1000research.8735.1. eCollection 2016.
Advances in gene therapy for muscular dystrophies.
Abstract
- PMID:
- 27594988
- PMCID:
- PMC4991540
- DOI:
- 10.12688/f1000research.8735.1
Public Health Genomics Knowledge Base (v1.2)
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MUSCULAR DYSTROPHY
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Last Updated: Sep 22, 2016
- FDA commissioner calls for Sarepta drug study to be retracted
StatNews, September 22, 2016 - Advances in gene therapy for muscular dystrophies.
Abdul-Razak Hayder et al. F1000Research 2016 5 - Current and emerging treatment strategies for Duchenne muscular dystrophy.
Mah Jean K et al. Neuropsychiatric disease and treatment 2016 121795-807 - FDA approves Sareptas controversial drug for Duchenne muscular dystrophy
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J Delviscio, StatNews, September 20, 2016 - Sarepta to charge $300K for Duchenne drug. We tried to be reasonable, CEO says
E Silverman, StatNews, September 19, 2016 - Large copy-number variations in patients with statin-associated myopathy affecting statin myopathy-related loci.
Stránecký V, et al. Physiological research / Academia Scientiarum Bohemoslovaca 2016 8 - Twenty-year follow-up of newborn screening for patients with muscular dystrophy.
Chung Jeffrey et al. Muscle & nerve 2016 Apr 53(4) 570-8 - "Target" and "Sandwich" Signs in Thigh Muscles have High Diagnostic Values for Collagen VI-related Myopathies.
Fu Jun, et al. Chinese medical journal 0 0 (15) 1811-1816 - Target resequencing of neuromuscular disease-related genes using next-generation sequencing for patients with undiagnosed early-onset neuromuscular disorders.
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Esterhuizen A I et al. South African medical journal = Suid-Afrikaanse tydskrif vir geneeskunde 2016 106(6) 10999 - Survey on genetic counseling and health management for symptomatic and asymptomatic female dystrophinopathy carriers in Japan today.
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Vears Danya F et al. Journal of genetic counseling 2016 May - The emerging role of viral vectors as vehicles for DMD gene editing
I Maggio et al, May 23, 2016
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