Thank you for donating!

You can donate using the following services.

NPUK Annual Family Conference

Our Friends

Here at NPUK you will regularly find us working with other like-minded organisations, such as those that support other connected rare diseases and other charities who share our main concerns and hopes. We have a track-record of successful collaborations with organisations that operate in line with our focus and commitment to improving the lives of those affected with Niemann-Pick disease(s) and other rare diseases.

We work and connect with other organisations both nationally and internationally, enabling us to develop a stronger, more united voice, to share knowledge and to access specialist information. Through our collaborations we will be able to achieve common goals, make a greater impact and accomplish more on behalf of those we support.

Our Friends

Support us

Latest news

01.11.21

Orphazyme provides regulatory update following Type A meeting with FDA on arimoclomol in Niemann-Pick disease type C

Progress made in understanding potential resolution of topics outlined in Complete Response Letter, including need for additional data to support NDA. FDA recommends the company provides supplemental information and analyses, and the FDA offers to have further interactions to identify a path to resubmission for arimoclomol in NPC...

Read more
View all news Subscribe