International Niemann–Pick Disease Alliance

Updates

  1. Orphazyme: Update #9 on the AIDNPC Clinical Programme

    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...

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  2. Update on the Clinical Trial of VTS-270: The NPC Study (August 2016)

    Update on the clinical trial of VTS-270: The NPC Study August 2016 Newsletter  Update on the Vtesse Clinical Trial of VTS-270: August was a very busy month here at Vtesse.  We continue to open up new sites and enroll...

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  3. Orphazyme: Update #8 on the AIDNPC Clinical Programme

    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...

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  4. 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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  5. 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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  6. 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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  7. 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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  8. 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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  9. 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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  10. 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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