International Niemann–Pick Disease Alliance
A constant smile and strong personality are just two traits of Isadora, a Brazilian girl who has became the first three year old in the world to receive enzyme therapy for Niemann-Pick type B disease. Upon hearing this news...
Marketwired Dec 21, 2016 8:00 AM ALACHUA, FL–(Marketwired – December 21, 2016) – CTD Holdings, Inc.(CTDH), a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease, today announced its filing of a Clinical Trial Application (CTA) with...
Pivotal Phase 2/3 Trial of Olipudase Alfa for Adult Patients with Acid Sphingomyelinase Deficiency (Niemann-Pick Disease Type B) to Begin in the UK Oxford, UK – 3 November, 2016 – Sanofi Genzyme, the specialty care global business unit of...
There is so much going on as of late; with various clinical trials beginning and/or expanding, new companies joining the fight against Niemann-Pick disease (Perlara and Novartis), and the partnership of Quinn Madeline and Baby Genes in order to create the first diagnostic carrier...
Clinical Trial Update: Orphazyme expands NPC study to three sites in the USA ( Download Here) Orphazyme’s clinical programme AIDNPC, investigating the orally administered small molecule arimoclomol as a treatment for Niemann-Pick disease type C, is close to opening...
Update #11 on the AIDNPC Clinical Programme (arimoclomol in treatment of Niemann-Pick disease type C) You can download the article here ...
HLN.BE REGIO The days have been tiring recently for the family from Tessenderlo, but they endured the biweekly trips to Amsterdam for the tests with a smile! Not exactly surprising when you learn that this year marks sixteen years...
You can download this press release here The Quinn Madeleine Foundation establishes first-ever Niemann-Pick diagnostic carrier screening program Aiming to eradicate fatal ASMD, The Quinn Madeleine Foundation has partnered with Baby Genes to create a diagnostic carrier screening program...
Perlara and Novartis join the fight against Niemann-Pick Disease: Perlara was founded in February 2014 and is the brainchild of molecular biologist Ethan Perlstein. Perlara is a biotech startup based in the USA, whose mission is to discover personalized...
Update from the International Niemann-Pick Disease Alliance (INPDA): The INPDA has been working hard throughout the year to facilitate progress for those affected by Niemann-Pick disease, by engaging in three key projects: the International Niemann-Pick Disease Registry (INPDR),...