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 …
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Niemann-Pick Type C News
IntraBio Completes Recruitment for IB1001 NPC Pivotal Clinical Trial
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 …
Science Update: Study on the Characteristics of swallowing in Niemann–Pick disease type C1
This newly released study represents the most extensive collection of swallowing evaluations in NPC1 to date. This large data series can serve as a foundation for NPC1 swallowing and speech domains assisting clinical management, quality of life decisions, and therapeutic outcomes for future clinical trials. Utilizing videofluoroscopic swallowing study (VFSS), also known as the modified …