Showing 4 of 4 recruiting trials for “lysosomal-disease”
Sirolimus for Leigh Syndrome
👨⚕️ Matthew Demczko, MD, Children's Hospital of Philadelphia📍 1 site📅 Started Feb 2025View details ↗
RecruitingNCT06967831 ↗
Drug Repurposing for Mitochondrial Disorders Using iPSCs Derived Neural Cells
👨⚕️ Markus Schuelke, MD, CHARITE - UNIVERSITAETSMEDIZIN BERLIN📍 2 sites📅 Started Mar 2020View details ↗
The International Registry for Leigh Syndrome
👨⚕️ Mary Kay Koenig, MD, The University of Texas Health Science Center, Houston📍 1 site📅 Started Jun 2015View details ↗
RecruitingNCT01780168 ↗
The NIH MINI Study: Metabolism, Infection, and Immunity in Inborn Errors of Metabolism
👨⚕️ Peter J McGuire, M.D., National Human Genome Research Institute (NHGRI)📍 1 site📅 Started Dec 2012View details ↗
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