ARTICLE | Distillery Therapeutics
Therapeutics: Mucolipin 1 (TRPML1; MCOLN1); inositol polyphosphate-5-phosphatase f (INPP5F)
July 21, 2016 7:00 AM UTC
Patient sample and cell culture studies suggest enhancing TRPML1 activity could help treat renal damage and failure associated with Lowe syndrome, which is caused by mutations in INPP5F. In primary hu...