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 …
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 …
KemPharm (now Zevra) 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 …