International Niemann–Pick Disease Alliance
The attached file contains a summary of the presentations and topics covered during the fourth biennial face to face meeting of INPDA members. This took place in Mainz, Germany, at the end of October 2015. Looking forward to 2017...
NEWS RELEASE – Copenhagen, 10 February 2016 Positive Review of Paediatric Investigation Plan (PIP) for AIDNPC . EMA’s Paediatric Committee (PDCO) recommends that the Committee for Medicinal Products for Human Use (CHMP) approve Orphazyme’s PIP for use of arimoclomol in treating patients suffering from Niemann-Pick disease type C (NP-C). The...
Now in its fifth year, Genzyme’s Patient Advocacy Leadership Awards (PAL Awards) program supports non-profit organizations that serve the lysosomal storage disorder (LSD) patient community. From Genzyme “As an independent voice, patient organizations play a vital role in providing...
A teleconference was hosted by Orphazyme 26th November 2015, to provide an update to patient organisations regarding the development of Arimoclomol in the AIDNPC programme. These calls will continue monthly – the next scheduled to take place 14th January...
The INPDA and its member groups have received the following update from Orphazyme, ApS: AIDNPC Clinical Programme change: Earlier initiation of Interventional study (CT-ORZY-NPC-002; Arimoclomol treatment). After valuable dialogue with regulators and patient organizations on the design of the Observational and Interventional...
“IMAGINE” follows the story Millie, who dreams of being a dancer yet suffers from Niemann-Pick Type C. Already viewed over 160,000 times on YouTube, we’re delighted that the English script has now been subtitled in 10 languages – Arabic,...
Copenhagen, 16 September 2015. A dedicated website to AIDNPC has been launched. The site describes Orphazyme’s clinical programme to investigate the orally available small molecule arimoclomol in Niemann-Pick disease type C. The website offers to NP-C patients a tool...
Orphazyme has announced a further update to its NP-C clinical programme. Arimoclomol will require the performance of a double-blinded clinical trial with a control group. The control group means that a third of patients enrolled will receive a placebo for...
Felix Schleuniger of Switzerland arrived in Banff, Alberta Canada on July 18th, 2015 at 1:00pm on schedule at the Banff Springs Hotel after biking 2,500 miles from Albuquerque, New Mexico USA on the Great Divide Mountain Bike Route for...
The “IMAGINE” short film, which follows the story of Millie, a little girl who dreams of being a dancer, yet suffers from Niemann Pick Type C, is now available with subtitles in Spanish, German, Polish and French – more to...