25.01.21
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.
Read more19.01.21
The government has just announced a new UK Disability Survey and we want to hear from as many people as possible. They are particularly keen to hear from disabled people, their carers, friends and family but views from the wider public are also very welcome. The survey will remain open until 23rd April, and all views will be used to shape the delivery of the plans we set out in the Strategy with those that we receive by 13th February informing its development.
Read more05.01.21
Home-based intravenous infusions of Trappsol® Cyclo™ for up to one year show improvement in disease features or disease stabilization...
Read more05.01.21
In recognition that many in our community are being impacted by the events of 2020, Trustees of NPUK and The Hollie Foundation, in recognition of their shared goals, have designated funds to create a new grant scheme for patients and families. The aim of this fund is to assist those experiencing hardship or unexpected expense, which threatens to impact their physical or mental well-being...
Read more
