International Niemann–Pick Disease Alliance
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...
Sanofi Genzyme Begins Pivotal Phase 2/3 Trial of Olipudase Alfa for Adult Patients with Acid Sphingomyelinase Deficiency CAMBRIDGE, Mass. – July 6, 2016 – Sanofi Genzyme, the specialty care global business unit of Sanofi, announced today that the first...
AIDNPC clinical programme – Arimoclomol in treatment of Niemann-Pick disease type C Please find below notes from a teleconference call that took place between patient organisations and Orphazyme on 29th JUNE 2016 SUMMARY 001 Study: – Recruitment has reached 33...
Press Release: FDA grants Fast Track designation for the Phase III clinical investigation of arimoclomol as a treatment of Niemann-Pick disease type C Copenhagen, Denmark, June 27, 2016. Orphazyme ApS announced today that the U.S. Food and Drug Administration...