The following article features a letter regarding the Study 301 Suspension from Sheila Talafous, Director of Advocacy Relations at Mallinckrodt Pharmaceuticals...Read more
We are pleased to inform you of a study opportunity available to NPC patients and their families:
Clinical Outcomes Solutions (COS), is a research company conducting research into Niemann-Pick type C (NPC) on behalf of a sponsor, Orphazyme A/S, a pharmaceutical company studying NPC...
Following the encouraging regulatory agency feedback, Orphazyme plans to introduce an Early Access
Program for NPC in the fall of 2019, to further accelerate access to treatment with arimoclomol for people
living with NPC...
This year the NPUK Interactive Workshop will take place on the 20th September at Wyboston Lakes, Bedfordshire - and will as ever be a fantastic opportunity for clinicians, health professionals and scientists working in the Niemann-Pick field to meet together under one roof...Read more