29.01.21
This webinar has been organised by patient organisations working in the field of Inherited Metabolic Diseases (IMD). We have realised from talking to IMD patients and families that there is some confusion about the COVID-19 vaccines...
Read more26.01.21
We are pleased to share the latest update from Cyclo Therapeutics entitled "Cyclo Therapeutics to Present on Its Clinical and Drug Development Program for Lead Candidate, Trappsol® Cyclo™, at WORLDSymposium 2021"...
Read more26.01.21
We are proud to have been nominated for the #EURORDISAwards2021 (EURORDIS Black Pearl Awards) this year, in the category of Visual and Audio Media Award...
Read more25.01.21
We are pleased to share the following article "A master protocol to investigate a novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasia” which describes the collaborative development of an innovative master protocol and primary outcome assessment to investigate N-acetyl-L-leucine as a potential treatment for three rare disorders, including Niemann-Pick disease type C (NPC)....
Read more20.01.21
We have received the following statement from Mallinckrodt Pharmaceuticals, stating their intention to discontinue clinical development of adrabetadex (VTS-270) for Niemann-Pick Type C1 disease (NPC), effective immediately. We understand that this will be very difficult news for many within our community; the NPUK team will be available if you would like to contact us: email info@npuk.org or telephone 0191 415 06 93.
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