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6 Studies found from all possible combinations of your search terms. Select a protocol that you wish to review.
Protocol Number Title Protocol Status Min-Max Age Institute Keywords
20-HG-0025A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Efficacy of ManNAc in Subjects with GNE MyopathyParticipants currently recruited/enrolled18-125 YearsNHGRIMyopathy
19-M-0079Evaluation of Novel PET Radioligands as Inflammatory Biomarkers in Rheumatoid Arthritis and MyositisParticipants currently recruited/enrolled18-99 YearsNIMH Myopathy
18-N-0083ASPIRO: A Phase 1/2/3, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) PatientsNo longer recruiting/follow-up only3-5 YearsNINDSMyopathy
16-DK-0036A Phase 1 Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ManNAc in Subjects with Primary Podocyte DiseasesCompleted Study; data analyses ongoing18-125 YearsNIDDKMyopathy
11-HG-0218A Natural History Study of Patients with GNE Myopathy and GNE-Related DiseasesParticipants currently recruited/enrolled4-80 YearsNHGRIMyopathy
00-N-0043Clinical and Molecular Manifestations of Inherited Neurological DisordersParticipants currently recruited/enrolled2-125 YearsNINDSMyopathy