International Niemann–Pick Disease Alliance
“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...
Copenhagen, Denmark, January 6, 2020 – Orphazyme A/S (ORPHA.CO), a biopharmaceutical company pioneering Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today announces the availability of an Early Access Program (EAP) in the United States (US) for its...
• 12-month data from open-label extension trial show sustained effect in reducing disease progression over two years • New genetic sub-group analysis recommended by US Food and Drug Administration supports robustness of results • Filling of New Drug Application...
Today, the INPDA received the attached letter (copied below), from Regan Sherman, Associate Director, Patient Advocacy Relations at Orphazyme. The letter provides the latest update regarding the introduction of an EAP programme for their investigational therapy, arimoclomol. Please do...
This week Cláubia Viegas Bender, co-founder of INPDA member group Niemann-Pick B-RS, completed her master’s degree with a project focused on ASMD Niemann-Pick disease type B. We are all hugely proud of her achievement, and we hope you will join...
Data show that Trappsol® Cyclo™, the Company’s proprietary hydroxypropyl beta cyclodextrin drug, reduces levels of trapped cholesterol in liver tissue of Niemann-Pick Disease Type C Patients GAINESVILLE, FL – (Businesswire) – December 16, 2019 – Cyclo Therapeutics, Inc. (OTCQB:...
Orphazyme receives Breakthrough Therapy Designation for arimoclomol in Niemann-Pick Disease Type C (NPC) • There are currently no US FDA-approved products for NPC • Orphazyme plans to file arimoclomol in the US in H1 2020 Copenhagen, Denmark, November 19,...
Publication describes favorable safety profile and efficacy in neurologic and other disease features in individual NPC patients following intravenous administration of drug Gainesville, FL – (Businesswire) – 31 October 2019 – Cyclo Therapeutics, Inc, (OTCQB: CTDH), a clinical stage...
Top line results are expected to be available in February 2020 GAINESVILLE, FL – (Businesswire) – October 22, 2019 – Cyclo Therapeutics, Inc. (OTCQB: CTDH), formerly CTD Holdings, Inc., a biotechnology company that develops cyclodextrin-based products for the treatment...
Announcement made in conjunction with October 19th Global Awareness Day for Niemann-Pick Disease sponsored by International Niemann Pick Disease Alliance GAINESVILLE, FL – (Businesswire) – October 18, 2019 – Cyclo Therapeutics, Inc. (OTCQB: CTDH), formerly CTD Holdings, Inc., a...