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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) …

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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 …

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Azafaros Announces IRB approval of 1st Site in U.S. for Phase 2 RAINBOW Study NPC and GM2

RAINBOW study design enables rapid advancement into the company’s planned Phase III efficacy trial Leiden, The Netherlands, May 22- 2023 – Azafaros B.V. today announced that the first US site for the Phase II RAINBOW study, led by Dr Marc Patterson of the Mayo Clinic in Minnesota, has received Institutional Review Board approval. The clinical …