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2016
- A Phase II Open-label, Multicenter Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) (website)
- A Phase IIa Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) (website)
- Phase I Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2 (website)
- Safety & Suitability of Dabigatran to Inhibit Thrombin in Scleroderma (website)
2015
- “A Phase 2, Double-blind, Randomized, Placebo-controlled Study to Investigate the Safety, Tolerability, and Efficacy of JBT-101 in Subjects With Dermatomyositis (website)
- Effect of atorvastatin on endothelial function and raynaud in diffuse scleroderma (website)
- First-in-human raavrh74.mck.galgt2 dmd clinical trial (website)
- Phase I/IIa Gene Transfer Clinical Trial for LGMD2D (Alpha-sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCA (website)
- TAMER: The Effect of Atorvastatin on Microvascular Endothelial Function and Raynaud in Early Diffuse Systemic Sclerosis (website)
- Vbp15, an innovative steroid-like intervention on dmd: vision-dmd (website)
2014
- Development of a strength training protocol in duchenne muscular dystrophy (website)
- Iron Therapy for Autosomal Dominant Hypophosphatemic Rickets: A Pilot (website)
- Treatment of dermatomyositis with ajulemic acid, a non-psychoactive cannabinoid (website)
2013
- Phase I/IIa Gene Transfer Clinical Trial for LGMD2D (Alpha-sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCA (website)
2011
- Calcitonin for Treating X-linked Hypophosphatemia (website)