Last Posted: Nov 30, 2019
- Muscular dystrophies
E Mercuri et al, The Lancet, November 2019 - Grants for Rare Disease Research
F Sankar, JAMA< November 19, 2019 - Genetic analysis of 62 Chinese families with Duchenne muscular dystrophy and strategies of prenatal diagnosis in a single center.
Zhang Jingjing et al. BMC medical genetics 2019 Nov 20(1) 180 - Gene-Editing Advance Puts More Gene-Based Cures Within Reach
F Collins, NIH Director Blog, November 5, 2019 - Clinical utility of RNA sequencing to resolve unusual GNE myopathy with a novel promoter deletion.
Chakravorty Samya et al. Muscle & nerve 2019 60(1) 98-103 - Vision of the Future of Medical Science
Francis Collins, Time, October 25, 2019 - New CRISPR tool has the potential to correct almost all disease-causing DNA glitches, scientists report
S Begley. Stat News, October 21, 2019 - Delays in diagnosis of Duchenne muscular dystrophy: an evaluation of genotypic and sociodemographic factors.
Counterman Kevin J et al. Muscle & nerve 2019 Oct - Diagnosis of Duchenne Muscular Dystrophy in Italy in the last decade: Critical issues and areas for improvements.
D'Amico Adele et al. Neuromuscular disorders : NMD 2017 May 27(5) 447-451 - Nano-Sized Solution for Efficient and Versatile CRISPR Gene Editing
NIH Director blog, September 17, 2019
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