News

Digital Annual Family Conference & Interactive Workshop 2020 (REGISTER NOW!)

We are excited to announce that registration for our very first Digital Annual Family Conference & Interactive Workshop on Niemann-Pick Disease, is now open!

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

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?