News

IntraBio Reports Positive Data from IB1001 Multinational Clinical Trial for the Treatment of Niemann-Pick disease Type C

Orphazyme announces U.S. FDA acceptance and Priority Review of New Drug Application for arimoclomol for Niemann-Pick disease Type C

NPUK Annual Family Conference & Interactive Workshop 2020 - FULL PROGRAMME

We are pleased to share the full programme for this year's Annual Family Conference & Interactive Workshop on Niemann-Pick Diseases, which will be the very first digital Conference we have held. Despite this we are sure it will be an informative, supportive, and entertaining weekend for all of the friends and families within our community...

Cyclo Therapeutics Inc. Announces Additional Efficacy Data from its Ongoing Phase I/II Trial using Trappsol® CycloTM Intravenously to Treat Patients with Niemann-Pick Disease Type C1 (NPC1)

Update on Adrabetadex - Dr. Steve Romano, Mallinckrodt’s Executive Vice President and Chief Scientific Officer

The following is an update on Adrabetadex from Dr. Steve Romano, Mallinckrodt’s Executive Vice President and Chief Scientific Officer...