Updates

Pfriegers Digest – 11th Issue

Azafaros announces positive topline Phase 2 study data with nizubaglustat

Azafaros announces positive topline Phase 2 study data with nizubaglustat in GM2 gangliosidosis and Niemann-Pick disease type C. Study primary endpoint met, with confirmed safety profile Investigators and Patient Organisations supportive of continued development Leiden, The Netherlands, 19 June...

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

Pfrieger’s Digest – 10th Issue

Zevra Therapeutics: FDA Extends Review Period for Arimoclomol NDA

IntraBio: NEJM Publishes Expert Perspective Editoral on IB1001

IntraBio Inc is pleased to announce that The New England Journal of Medicine (NEJM) has published a Science Behind the Study Expert Perspective Editorial: N-Acetyl-l-Leucine and Neurodegenerative Disease. The Editorial reports on the broad potential of N-acetyl-L-leucine (IB1001) as...

NEJM publishes IB1001-301 Trial Results

IntraBio Inc is pleased to announce that The New England Journal of Medicine (NEJM) has published the detailed results of the Phase 3, Pivotal Trial with N-acetyl-L-leucine (IB1001) for the treatment of Niemann-Pick disease Type C (NPC). New England...

Azafaros’ Phase 2 RAINBOW study, evaluating nizubaglustat in GM2 and NPC patients, is now fully enrolled

Cyclo Therapeutics Announces Positive Outcome from Type C Meeting with the U.S. FDA Discussing Trappsol® Cyclo™ Clinical Program for the Treatment of Niemann-Pick Disease Type C1

Introducing the INPDA International Niemann-Pick Disease Alliance Executive Committee

Introducing the newly elected INPDA International Niemann-Pick Disease Alliance Executive Committee (from left to right); Sarah Janetta, Executive Communications Officer, Joslyn Crowe, Vice President, Sandy Cowie, President and Toni Mathieson, Executive Secretary. We are so proud of the team we have...