December 11, 2023 Leiden, The Netherlands, 11 December 2023 – Azafaros B.V. today announces that it has exceeded the target number of patients in the enrollment of its Phase 2 clinical trial, RAINBOW (NCT05758922), treating patients with severe rare metabolic diseases. The success in enrollment is a major milestone for Azafaros’ lead asset, nizubaglustat, which is …
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Niemann-Pick Type C News
IV Adrabetadex (cyclodextrin) Trial for Neonatal Liver Disease Started
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 …
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) …