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Dr. Randy Chandler is an associate investigator in the Organic Acid Research Section of the Metabolic Medicine Branch in NHGRI.
… methylmalonic acidemia (MMA), propionic acidemia (PA), and Niemann-Pick Disease, Type C1. His preclinical proof-of-principal … labs, he led efforts to develop AAV gene therapy to treat Niemann-Pick Disease, Type C1 disease, a rare and lethal …
News Release
Principal investigator-mentors guide new scientists to their next chapter.
… He investigated ways to track the severity and onset of Niemann Pick Type C disease (NPC), a rare lysosomal storage disorder …