Researchers from St. Louis Children’s Hospital at Washington University School of Medicine are enrolling infants 0-6 months with diagnosed NPC disease and evidence of liver disease. The purpose of the study is to learn how well adrabetadex can reduce liver damage in infants diagnosed with NPC disease. The study has an initial 6-week phase during …
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Niemann-Pick Type C News
Bloomsbury Genetic Therapies Receives Rare Pediatric Disease Designation from FDA for Gene Therapy Treatment of NPC
21 August 2023 – RPDD granted in the United States – London, UK, 21 August 2023 – Bloomsbury Genetic Therapies Limited, a clinical-stage biotechnology company developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies, announced today that the U.S. Food and Drug Administration (FDA) …
IntraBio Announces Successful NPC Phase III Trial
IntraBio Announces Positive Pivotal Trial Results of IB1001 for the Treatment of Niemann-Pick Disease Type C OXFORD, UK / ACCESSWIRE / June 29, 2023 / IntraBio Inc today announced positive topline results from the pivotal, Phase III IB1001-301 clinical trial (NCT05163288), showing IB1001 significantly improved symptoms, functioning, quality of life, and cognition in pediatric and …