International Niemann–Pick Disease Alliance
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...
Available for download, here.
Manoir de Clénord, Chambord, France October 18-19, 2018 The following report summarizes discussions at the NP-C Gene Therapy Activation Meeting (GAM), a first-of-its-kind satellite meeting to the bi-annual Loire-Valley Meeting (LVM) hosted by the German and Swiss Niemann-Pick family...
ORPHAZYME REPORTS ENCOURAGING ARIMOCLOMOL CLINICAL TRIAL TOP-LINE DATA IN NIEMANN-PICK DISEASE TYPE C (NPC) Arimoclomol was well-tolerated in patients with the rare disease NPC The NPC Phase II/III trial top-line results show a treatment benefit of arimoclomol over placebo...
In the following video INPDA President and Former NPUK Trustee Jim Green defines what exactly the International Niemann-Pick Disease Registry (INPDR) is, and why it is needed: “…the International Niemann-Pick Disease Registry aims to collect data about the disease...
Founder and Chair of the Taiwan Niemann-Pick Disease Patient Group, Chin Huei Wu (巫錦輝), gave a rousing presentation at the NPUK Annual Family Conference & Interactive Workshop on Niemann-Pick Disease (2017) in which he gave the story of his...
Due to changes in data protection legislation, soon the International Niemann-Pick Disease Alliance (INPDA) won’t be able to contact you about any of our services or activities unless you explicitly give your consent. In other words, if you would like to...
ALACHUA, FL – (Marketwired) – April 30, 2018 – CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of disease with unmet medical need, today announced that FDA has authorized its...
ALACHUA, FL — (Marketwired) — 04/23/18 — CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of disease with unmet medical need, today announced that it has closed a private placement...