International Niemann–Pick Disease Alliance
Data cleaning underway, top line results expected the first quarter of 2020 GAINESVILLE, FL – (Businesswire) – February 11, 2020 – Cyclo Therapeutics, Inc. (OTCQB: CTDH), a biotechnology company that develops cyclodextrin-based products for the treatment of Niemann-Pick Disease...
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) – February 5, 2020 – Cyclo Therapeutics, Inc. (OTCQB:...
Positive topline results demonstrated by olipudase alfa, first and only investigational therapy in late-stage development for acid sphingomyelinase deficiency · Acid sphingomyelinase deficiency (ASMD) is a rare, progressive and potentially life-threatening disease for which no treatments are approved PARIS...
OXFORD, UK / ACCESSWIRE / January 17, 2020, / IntraBio Inc, a late-stage biopharmaceutical company, is pleased to share that it has completed enrollment for its IB1001-201 Clinical Trial. IB1001-201 investigates N-Acetyl-L-Leucine (IB1001) for both the symptomatic, and longterm,...
OXFORD, UK / ACCESSWIRE / January 15, 2020 / IntraBio Inc., a late-stage biopharmaceutical company, is pleased to share that the Extension Phase for the IB1001-201 Clinical Trial has been approved in all European countries where the trial is...
“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...