Updates

Orphazyme presents 36-month data supporting durable response to arimoclomol during Parseghian Scientific Conference for NPC Research

INPDA Community Webinar Series: Cyclo Therapeutics – Phase 3 Trial

Join us on Wednesday June 30th at 7pm BST to hear an update direct from Cyclo Therapeutics regarding their Phase 3 Clinical trial of Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C: The INPDA is pleased to...

Orphazyme provides regulatory update from FDA on arimoclomol for Niemann-Pick disease type C

Orphazyme A/S Company announcement No. 16/2021 Inside information Company Registration No. 32266355 Copenhagen – June 18, 2021 – Orphazyme A/S (ORPHA.CO; ORPH), a late-stage biopharmaceutical company pioneering the heat shock protein response for the treatment of rare diseases, today...

Orphazyme: NPC Community Notification

Cyclo Therapeutics Appoints Lori McKenna Gorski as Global Head of Patient Advocacy

INPDA Spotlight on Fundación Niemann-Pick de España (FNP)

Sometimes in such a big organisation as the INPDA, our various member groups can be overshadowed, but we want to show just how important each and every member group is in making the INPDA work. In the INPDA Spotlight this...

Cyclo Therapeutics Announces Design of Pivotal Phase 3 Study Evaluating Trappsol® Cyclo™ in Niemann-Pick Type C1

Cyclo Therapeutics Inc: Update (13/4/21)

INPDA Spotlight on VML France Niemann Pick Group

Sometimes in such a big organisation as the INPDA, our various member groups can be overshadowed, but we want to show just how important each and every member group is in making the INPDA work. In the INPDA Spotlight this...

Pfrieger’s Digest – 4th Issue

“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...