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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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Bloomsbury Genetic Therapies Outlines Pre-Clinical Data on CNS Programs including NPC

London, UK, 18 May 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 pre-clinical data from two of its brain-targeted programs, BGT-INAD and BGT-NPC for the treatment of Infantile Neuroaxonal Dystrophy …

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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 …