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2016
- An Exploratory Phase II Study to Determine the Tolerability, Safety, and Activity of a Novel Vasopressin 1a Receptor Antagonist (SRX246) in Irritable Subjects With Huntington’s Disease (HD) (website)
- Primary Prevention of Stroke in Children With Sickle Cell Disease in Sub-Saharan Africa II (website)
2015
- A Phase I Study of Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy (website)
- Exercise intervention for slowing HD progression (website)
- The effect of vitamin d3 on markers of oxidative stress in boys with x-linked ALD (website)
- Tolerability of srx246 in Huntington’s disease patients (website)
2014
- A Phase II Trial of Rituximab In Myasthenia Gravis (website)
2013
- A Dose-Escalation, Safety And Feasibility Study Of Enteral Levetiracetam For Seizure Control In Pediatric Cerebral Malaria (website)
- Duchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid Regimen (website)
- Phase I/IIa Gene Transfer Clinical Trial for LGMD2D (Alpha-sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCA (website)
- Phase 2 Clinical Trial of SGS-742 Therapy in Succinic Semialdehyde Dehydrogenase Deficiency (website)
- Sickle Cell Disease – Stroke Prevention in Nigeria Trial (website)
- Transcranial Magnetic Stimulation (TMS) in Spino-Cerebellar Ataxia (website)