International Niemann–Pick Disease Alliance
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...
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...