International Niemann–Pick Disease Alliance
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),...
PRESS RELEASE: (Download) Update #10 on the AIDNPC clinical programme (Arimoclomol in treatment of Niemann-Pick disease type C) Conference call with patient organizations (22 SEPTEMBER ’16) SUMMARY 002 Study: Recruitment into the 002 Interventional Study 002 Study:...
CTD Holdings Announces FDA Acceptance of Investigational New Drug Application for Treatment of NPC With Trappsol(R) Cyclo(TM) ALACHUA, FL–(Marketwired – September 06, 2016) – CTD Holdings, Inc. (CTDH), a biotechnology company that develops cyclodextrin-based products for the treatment of...
PRESS RELEASE: (Download) Update #9 on the AIDNPC clinical programme (Arimoclomol in treatment of Niemann-Pick disease type C) Conference call with patient organizations (26 AUGUST ’16) SUMMARY 002 Study: Recruitment into the 002 Interventional Study 002 Study: Patient...
PRESS RELEASE: (Download) Update #8 on the AIDNPC clinical programme (Arimoclomol in treatment of Niemann-Pick disease type C) Conference call with patient organizations (28 JULY ’16) SUMMARY: 001 Study: Recruitment has reached 33 patients enrolled at 12 sites...