International Niemann–Pick Disease Alliance
My name is Raphaël Beaugillet, I come from France and I am a friend of Alix (age 36) and Zita (age 30) both of whom are affected by the ultra-rare Niemann-Pick-C disease (NP-C). I will participate in the Road...
ALACHUA, FL – (Globe Newswire) – March 14, 2019 – CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of disease with unmet medical need, announced today that the company has...
OXFORD, UK, March 11, 2019– IntraBio Inc., a late-stage biopharmaceutical company, today announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved its Clinical Trial Authorisation (CTA) Application for Clinical Trial IB1001-201 with its lead compound...
CTD Holdings Announces Grant to U.S. Non-Profit Organization to Support Patient Participation in Informational Meetings with Regulators Support is part of CTD’s efforts to recognize Rare Disease Day 2019 ALACHUA, FL – (Globe Newswire) – February 28, 2019 –...
JJ was born with an enlarged spleen and liver and it wasn’t until the age of three that he was diagnosed with Acid Sphingomyelinase Deficiency (ASMD), also known as Niemann-Pick disease type B. In the following video he describes...
Orphazyme A/S Company announcement No. 01/2019 Company Registration No. 32266355 Primary endpoint shows 74% reduction in disease progression after 12 months compared to placebo control (p-value =0.0506) Biomarker results demonstrate statistically significant biological response to treatment Orphazyme to engage...
CTD Announces Partnership with United BioSource LLC to support the Extension Protocol for its Phase I Clinical Trial in the United States Collaboration will allow patients in Extension Protocol to receive CTD’s drug intravenously at home ALACHUA, FL –...
Annual conference offers opportunities for academic researchers and industry representatives to exchange information on novel therapeutic interventions. ALACHUA, FL – (Globe Newswire) – January 09, 2019 – CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops...
Sanofi Genzyme’s Global Project Team would like to announce that our pivotal registration study (ASCEND Study) for adult patients with chronic, systemic acid sphingomyelinase deficiency (ASMD) Types B and A/B (or intermediate type) and our pediatric study (ASCEND-Peds) also...
Copenhagen, December 10, 2018 – Orphazyme A/S, a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announced that the Company expects to announce the results of the full data set for the clinical Phase...