OXFORD, UK / December 08, 2022 / IntraBio Inc announced today that it has completed recruitment for its Pivotal Trial, Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study (IB1001-301 – NCT05163288). Enrolment for IB1001-301 commenced in September 2022. Patients were screened across 13 multinational trial sites …
6th Annual DART To The Finish Charity Walk * Sat. * Sept. 30, 2023 * 9:00a
Gather your friends and family and join is for another invigorating 2-mile walk with beautiful views of Long Island Sound around Tod’s Point in Old Greenwich, and help us raise much needed funds to find a cure for Niemann-Pick type C disease. No beach pass, no problem. DART has you covered! The morning always makes …
DART to The Finish Charity Walk Raises $175,000
The fifth annual DART to The Finish charity walk benefitting Dana’s Angels Research Trust (DART) took place Saturday, September 24, 2022, at Greenwich Point Park in Old Greenwich, and virtually across the country. More than 300 participated and helpedraised more than $175,000, which will go towards supporting crucial research into better treatments, clinical trials and …
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 …
Azafaros Receives FDA’s IND Clearance and Fast Track Designation
Azafaros B.V. announced in January 2023 that it Investigational New Drug (IND) clearance from the United States Food and Drug Administration (FDA) to conduct a clinical Phase 2 trial for its lead asset, AZ-3102, for the treatment of GM2 gangliosidosis (GM2) and Niemann-Pick disease type C (NP-C). In addition to the IND clearance, the FDA …