News

Orphazyme completes rolling submission of New Drug Application to U.S. FDA for arimoclomol for Niemann-Pick disease Type C

Mallinckrodt Pharmaceuticals Statement: Adrabetadex for NPC-1

Please take a moment to read this important announcement from Mallinckrodt Pharmaceuticals in regard to the clinical trial of Adrabetadex for NPC-1. We recognise that this may be a surprising and disappointing development for our community as a whole. Please be assured that we are advocating for further, clearer information and will follow up with our response and next steps shortly.

Sandra Cowie, President, INPDA
Joslyn Crowe, Executive Director, NNPDF
Toni Mathieson, Chief Executive, NPUK

UK LSD Collaborative: Survey 2020

Revised Guidance on Shielding (NPUK Statement)

With the risk of catching coronavirus steadily decreasing, the UK Government yesterday announced changes to the future of the shielding programme. From 6 July those shielding will be able to meet outdoors, in groups of up to six people from outside their household, with social distancing. If you live alone (or are a lone adult with dependent children under 18), you will be able to form a support bubble with another household. From 1 August, many people will no longer need to shield, and the advice will be that you can visit shops and places of worship, but you should continue to maintain social distancing whenever possible...

Can you help to support trainees in inherited metabolic disease and lysosomal storage disorders?