International Niemann–Pick Disease Alliance
Available for download in English, French, German, Italian, Polish, and Spanish Dear Niemann-Pick type C Community, We are pleased to inform you that on November 16th Orphazyme A/S was listed on the Nasdaq OMX exchange in Copenhagen through an...
Medical geneticists at the Clinical Research Center of the Hospital de Clinicas in Porto Alegre are betting on an unprecedented treatment for a rare disease, known as ASMD Niemann-Pick disease type B. The disease has no cure, and causes...
We are happy to introduce the International Niemann-Pick Disease Registry (INPDR)’s official Facebook page – which will provide any and all relevant updates pertaining to the INPDR. But, what is the INPDR? The INPDR is a unifying force that has...
Update #17 on the AIDNPC Clinical Programme (arimoclomol in treatment of Niemann-Pick disease type C) Available for download in English, French, German, Italian, Polish & Spanish Interim information bulletin (following the Sept. 28th call) SUMMARY 001 Study: Conclusions and...
Two New Israeli Sites Now Recruiting Patients in Multicenter European Trial Full press release available for download here ALACHUA, FL – (Marketwired) – September 18, 2017 – CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage biotechnology company developing cyclodextrin-based...
We are sorry to share the sad news that Ara Parseghian, passed away yesterday. Ara was a former title winning coach of Notre Dame and founded the Ara Parseghian Medical Research Foundation, which has raised over $45million for research...
In the following video (provided courtesy of NPUK) Dr. Ahad A. Rahim: Lecturer in Translational Neuroscience & UCL Excellence Fellow in Neurological Disease Modelling, gives an excellent breakdown of what gene therapy is, and how it holds a lot...
We are happy to announce the Niemann Pick Symposium, pre-conference satellite meeting to the 12th Annual ICORD Conference and 6th China Rare Disease Summit, will take place this year on the 7th September at the JW Mariott Hotel Beijing. The event,...
Following an update meeting with Genzyme regarding the Phase 2/3 Olipudase Alfa trial in adult patients (18+ years old) with ASMD (Niemann Pick Disease Type B), we are pleased to provide an update on the status of the Sanofi...
CTD Holdings Ships Additional Trappsol® Cyclo™ to Brazil Trappsol® Cyclo™ Is the Exclusive Form of Hydroxypropyl Beta Cyclodextrin Purchased by the Government of Brazil for the Compassionate Treatment of Niemann-Pick Disease Type C Available for download, here ALACHUA, FL...