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 …
Year
Showing: 4 - 6 of 6 RESULTS
2022
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 …
DART To The Finish Charity Walk for NPC 2022 – Saturday * Sept. 24, 2022 * 8:00a
Gather your friends and family and join us for this year’s invigorating 2.0 mile walk around beautiful 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. We’re expecting a wondrful in-person event, but if …