International Niemann–Pick Disease Alliance
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...
Last week, our campaign to improve diagnosis of Niemann Pick Type C (NP-C) went on the road to Lyon, France, to take part in the SSIEM’s (Society for the Study of Inborn Errors of Metabolism) Annual Symposium. Representatives from...
Calporta Therapeutics is developing small molecule agonists of TRPML1 for the treatment of Niemann-Pick C Disease (NPC) and other lysosomal storage diseases. Calporta’s approach is based on research from the lab of Haoxing Xu, Ph.D., at the University of...
Genzyme Initiates Phase 1/2 Clinical Trial to Evaluate Olipudase Alfa in Pediatric Patients: First patient dosed with investigational enzyme replacement therapy designed to treat the nonneurological manifestations of ASMD which characterize Niemann-Pick disease type B. Monday, June 15, 2015...
“IMAGINE” follows the story of Millie, a little girl who dreams of being a dancer, yet suffers from Niemann Pick Type C; an extremely rare, incurable disease which causes neurological decline and dementia. To watch the film, visit http://www.imagineshort.com/ 17...
IMAGINE follows Millie, a little girl suffering from NP-C. Imagine has been developed in collaboration with the Hope for Hollie Foundation, NPUK and the INPDA, to raise awareness of the disease. We can’t wait to share it!
Orphazyme’s ‘Observational’ study for NP-C has been registered and made available on www.clinicaltrials.gov. The trial identifier is NCT02435030. Confirmed sites and contact details are provided. https://clinicaltrials.gov/ct2/show/NCT02435030?term=arimoclomol&rank=5 You can find further information on Orphazyme’s website www.orphazyme.com