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Neurology|April 11, 2001
Familial cerebellar ataxia with muscle coenzyme Q10 deficiencyO Musumeci, A Naini, A E Slonim, et al.
American Journal of Public Health|August 29, 2002
Improving the health of workers in indoor environments: priority research needs for a national occupational research agendaMark J Mendell, William J Fisk, Kathleen Kreiss, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|November 1, 2014
Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primatesKathrin Meyer, Laura Ferraiuolo, Leah Schmelzer, et al.
Genes & Development|November 15, 2014
Tumor suppression by miR-26 overrides potential oncogenic activity in intestinal tumorigenesisLauren R Zeitels, Asha Acharya, Guanglu Shi, et al.
Neurology|April 26, 2001
A randomized efficacy and safety trial of oxandrolone in the treatment of Duchenne dystrophyG M Fenichel, R C Griggs, J Kissel, et al.
Pediatric Neurology|September 26, 2020
Natural History of Steroid-Treated Young Boys With Duchenne Muscular Dystrophy Using the NSAA, 100m, and Timed Functional TestsNatalie F Miller, Lindsay N Alfano, Megan A Iammarino, et al.
Nature|July 22, 2014
miR-34a blocks osteoporosis and bone metastasis by inhibiting osteoclastogenesis and Tgif2Jing Y Krzeszinski, Wei Wei, HoangDinh Huynh, et al.
Orphanet Journal of Rare Diseases|April 24, 2015
The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug developmentEmma Heslop, Cristina Csimma, Volker Straub, et al.
Molecular Therapy. Methods & Clinical Development|January 20, 2018
Safety and Efficacy of AAV Retrograde Pancreatic Ductal Gene Delivery in Normal and Pancreatic Cancer MiceKayla A Quirin, Jason J Kwon, Arafat Alioufi, et al.
Cell|May 27, 2014
An essential mesenchymal function for miR-143/145 in intestinal epithelial regenerationRaghu R Chivukula, Guanglu Shi, Asha Acharya, et al.
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