NIH Clinical Center: Search the Studies

7 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
001737-NR	A Single-Center Prospective Natural History Study of RYR1-Related Disorders	Recruitment has not started	7-100 Years	NINR	Myopathy
001625-HG	Prospective Study of the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders	Participants currently recruited/enrolled	0-100 Years	NHGRI	Myopathy
20-HG-0025	A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Efficacy of ManNAc in Subjects with GNE Myopathy	Participants currently recruited/enrolled	18-125 Years	NHGRI	Myopathy
19-M-0079	Evaluation of a Novel PET Radioligand as an Inflammatory Biomarker in Musculoskeletal Conditions	Participants currently recruited/enrolled	18-99 Years	NIMH	Myopathy
18-N-0083	ASPIRO: 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) Patients	No longer recruiting/follow-up only	3-5 Years	NINDS	Myopathy
11-HG-0218	A Natural History Study of Patients with GNE Myopathy and GNE-Related Diseases	Participants currently recruited/enrolled	4-80 Years	NHGRI	Myopathy
00-N-0043	Clinical and Molecular Manifestations of Inherited Neurological Disorders	Participants currently recruited/enrolled	2-120 Years	NINDS	Myopathy