International Niemann–Pick Disease Alliance
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...
We are writing to inform you that the CT-ORZY-NPC-002 trial activities as well as ongoing early-access and named-patient programs for arimoclomol in NPC will continue for the current time. As you may have seen, Orphazyme participated in an ad-hoc...
– Second Orphan Drug Designation Supports Azafaros’ Strategy to Develop AZ-3102 as a Disease Modifying Treatment in a Range of Severe Rare Inherited Metabolic Disorders – Leiden, The Netherlands, March 24, 2022 – Azafaros B.V. today announced that the...
March 22, 2022 06:54 ET | Source: ORPHAZYME A/S Orphazyme A/S Company announcement No. 13/2022 Inside information www.orphazyme.com Company Registration No. 32266355 Decision to withdraw the European Marketing Authorisation Application comes ahead of scheduled final vote on the application later this month Orphazyme intends to request Type C Meeting with...
The International Niemann-Pick Disease Alliance (INPDA) has organised a letter (see below) voicing the perspective and concerns of the NPC community, which will be sent to the European Medicines Agency (EMA) next week. Families also have the opportunity to...