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 …
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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 …
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 …