International Niemann–Pick Disease Alliance
Want to join the NNPDF Family Support & Medical Conference but can’t be there in person? VIRTUAL Conference registration is available! Cost is per family (includes multiple devices) and includes main sessions. Check out the agenda at t.ly/agenda2022 for all LiveStream...
Xenpozyme® (olipudase alfa) approved by European Commission as first and only treatment for ASMD Paris, June 28, 2022. The European Commission (EC) has approved Xenpozyme® (olipudase alfa) as the first and only enzyme replacement therapy for the treatment of non-Central Nervous System (CNS) manifestations of...
IntraBio Inc. Pivotal Trial Announcement: IB1001-301, “Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study.” We are pleased to share the news that recruitment to IntraBio’s Pivotal trial: IB1001-301 “Effects of...
May 2022 To The NPC1 Community, Happy May. It’s been a little while since we provided our last communication back in February and we’re excited to share three important updates on our progress. We have completed our first meeting...
Dear Members of the NPC Community, My name is Scott Fine and I have been CEO of Cyclo Therapeutics since 2015. Our company is enrolling a Phase 3 study of a potential treatment for NPC. The study, called “TransportNPC”,...
Recommendation based on positive results from two clinical trials in which Xenpozyme provided improvement across multiple non-CNS clinical manifestations of ASMD in pediatric and adult patients ASMD is a rare, progressive, and potentially life-threatening disease with no...
Orphazyme A/S in restructuring Company announcement No. 24/2022 Inside information www.orphazyme.com Company Registration No. 32266355 KemPharm to acquire Orphazyme assets, including those relating to the development and approval of arimoclomol, for a total of USD 12.8 million in cash and...
“Pfrieger’s Digest” is written by Frank Pfrieger of Niemann-Pick Selbsthiifegruppe (Germany) to provide an overview of the latest advances in Niemann-Pick disease based on recent scientific publications. Frank Pfrieger is group leader at the Institute of Cellular and Integrative Neurosciences in...
We are pleased to be able to share the following collaborative webinar entitled The impacts of olipudase alfa on pediatric patients with ASMD and their families: Results of an international survey, which was hosted by the International Niemann-Pick Disease Alliance (INPDA),...
DOWNLOAD THE PDF VERSION Xenpozyme® (olipudase alfa) approved in Japan, first and only approved therapy indicated to treat acid sphingomyelinase deficiency Xenpozyme represents first Sanofi therapy to be approved under the SAKIGAKE ‘fast-track’ designation Approval based on positive results from two separate clinical trials...