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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 …

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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 …

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KemPharm to acquire Orphazyme and pursue FDA approval for Arimoclomol

Arimoclomol treatment. On May 15, 2022, KemPharm, Inc., a specialty pharmaceutical company focused on rare central nervous system diseases like NPC, announced its acquisition from Orphazyme of Arimoclomol. This action supports the confidence of NPC clinicians in the benefit of Arimoclomol. “We believe the efficacy signal for Arimoclomol in NPC is convincing and that there …