The National Institutes of Health (NIH), a part of the U.S. Department of Health and Human Services, is the nation’s medical research agency — making important discoveries that improve health and save lives.
Clinical Trials.gov – Natural History Study at NIH
NIH Protocol Details – A Phase 1/2, Open-Label, Single Dose Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of SEL-302 (MMA-101 following administration of SEL-110) in Pediatric Subjects with Mut Subtype Isolated Methylmalonic Acidemia (MMA)
The reiMMAgine trial is a Phase 1/2 study which aims to investigate how children diagnosed with mut subtype isolated MMA respond to the gene therapy SEL-302 (a combination of SEL-110 and MMA-101), and if the study medication will help with their symptoms.
Clinical Trials.gov – Natural History Study on Propionic Acidemia
Articles from Previous OAA Newsletters
MMA Study at the NIH
PA Study at the NIH
About the National Institutes of Health (NIH): NIH, the nation’s medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases.
For more information about NIH and its programs, visit www.nih.gov.