International Niemann–Pick Disease Alliance
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...
CTD Holdings Enrolls First Patient in European Phase I/II Clinical Trial of Trappsol® Cyclo™ for Treatment of Niemann-Pick Disease Type C Company Expects Final Data from Trial by End of 2018 Alachua, FL – (Marketwired) – June 21, 2017...
The Annual VML Members’ Weekend was held from Friday 26 May to Sunday 28 May 2017 at the Domaine de Chalès in Nouan-le-Fuzelier (41) France – as ever it was an occasion filled with fun as the community came...
CTD Holdings Announces Appointments of Two Patient Liaisons to Enhance Outreach to Niemann-Pick Disease Type C Families and Caregivers Liaisons to Provide Counsel to Patient Families Around Recently Initiated Phase I and Phase I/II Clinical Trials ALACHUA, FL –...
The Pactimo online charity shop, which seeks to support the International Niemann-Pick Disease Alliance (INPDA), is now open! Summer is almost in full swing so why not have a look at our biking suits or sports T-shirts for either...
(Available for Download in English, German, French, Spanish, Italian, and Polish) Interim Information Bulletin SUMMARY 002 Study: All patients have now started treatment with study medicine Orphazyme to present at Spanish NPC Association on June 24th The...
The 21st Niemann-Pick Sielbstilfegruppe family support meeting took place in Kassel last weekend on Saturday 27th May. This was the 17th year that it has been held in the same location, one ideally positioned in the geographical centre of...