International Niemann–Pick Disease Alliance


  1. Sanofi Genzyme Begins Pivotal Phase 2/3 Trial for ASMD

    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...

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  2. Update on AIDNPC clinical programme – notes from teleconference

    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...

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  3. FDA grants fast track designation for Arimoclomol Phase III clinical investigation

    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...

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  4. Orphazyme begins dosing of NP-C patients in arimoclomol AIDNPC interventional study

    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...

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  5. INPDA Clinical Trials Statement

    There are two clinical trials for NP-C, and two for ASMD (NP-B) recruiting at present. The last clinical trial for NPD, for Miglustat, was almost 10 years ago. Opportunities for finding these long hoped for treatments don’t come along...

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  6. FDA gives clearance to arimoclomol study for treatment of NP-C

    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,...

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  7. Vtesse Advances Phase 2b/3 Clinical Trial of VTS-270 for NP-C

    Vtesse Advances Phase 2b/3 Clinical Trial of VTS-270 in Niemann-Pick Type C1 Disease with Dose Selection for Evaluation in Second and Final Portion of Trial and Expansion into Europe Ten clinical sites now enrolling patients in the United States,...

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  8. Newborn screening test developed for NP-C

    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)

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  9. Update from Vtesse May 2016

    Vtesse provided the following update today 04 May 2016: Click this link to view the full newsletter: Vtesse newsletter May 2016 Update on the Vtesse Clinical Trial of VTS-270 Vtesse is pleased to announce that Part A is fully enrolled. Part...

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  10. AIDNPC – summary of teleconference minutes 31st March 2016

    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...

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