Posts filed in: "News"

NPUK Frequently Asked Questions (FAQs) | NICE Xenpozyme (Olipudase Alfa) Decision

Calling all NPC Patients & Families: your help is needed!

Jackson Pountney, a PhD student at Aston University in Birmingham, needs your help. Jackson is undertaking a research project that will help us to better understand the impact of NPC on quality of life. This important work will help to advance understanding of NPC and support access to treatments in the UK and beyond...

Azafaros: Completion of 12-week Phase 2 RAINBOW study evaluating lead asset nizubaglustat in rare disease patients

NPUK Statement: NICE Olipudase Alfa Decision

Yesterday, we were greatly disappointed to learn that the UK's National Institute for Health and Care Excellence (NICE) has decided not to recommend Sanofi's Xenpozyme (olipudase alfa) as a treatment for acid sphingomyelinase deficiency (ASMD; Niemann-Pick Disease) in people with type AB or type B...

2024 Regional Family Days: Save the Dates!

We're pleased to announce this year's Regional Family Days, designed to bring our community together for support and solidarity. Here's the schedule...