International Niemann–Pick Disease Alliance
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...
Press Release: Copenhagen, Denmark, 15 June 2016. Orphazyme announced today that the first two patients have been dosed with arimoclomol in the Phase III ‘AIDNPC’ Niemann-Pick disease type C clinical trial programme. The first two patients in the NPC-002...
Press Release: Copenhagen, Denmark, June 06, 2016 Orphazyme has received a “Study May Proceed” letter from the FDA on Orphazyme’s protocol for arimoclomol as a new treatment for Niemann-Pick disease type C (NP-C), the AIDNPC intervention study. This makes the US IND1 effective,...
A study led by Washington University School of Medicine in St Louis, USA, describes a newborn screening test that identifies newborns with NP-C. The research appears May 4, 2016, in the journal Science Translational Medicine. 2016-05-newborn-screening-rare-deadly-neurological (1)
A teleconference was hosted by Orphazyme on 31st March 2016, to provide an update to patient organisations regarding the development of Arimoclomol in the AIDNPC programme. The next call is scheduled to take place 28th April 2016. In summary, the call...