International Niemann–Pick Disease Alliance

Updates

  1. 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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  2. Vtesse present Phase 1/2 Clinical Data showing slowing of disease progression in NPC1

    Press Release Vtesse, Inc. Announces Phase 1/2 Clinical Data Showing Slowing of Disease Progression from VTS-270 Treatment for Niemann-Pick Type C1 Disease GAITHERSBURG, Md., March 4, 2016 /PRNewswire/ — Vtesse, Inc. announced today data from a Phase 1/2 clinical trial demonstrating...

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  3. Summary of INPDA’s face to face meeting, Mainz, October 2015

    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...

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  4. Positive Review of Paediatric Investigation Plan (PIP) for AIDNPC

    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...

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  5. INPDA receives Patient Advocacy Leadership Award

    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...

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  6. Vtesse, Inc. Announces FDA’s Granting of Breakthrough Therapy Designation for VTS-270 in Niemann-Pick Type C1 Disease

    Vtesse, Inc. Announces FDA’s Granting of Breakthrough Therapy Designation for VTS-270 in Niemann-Pick Type C1 Disease ⎯ Kevin B. Johnson, Ph.D., Joined Vtesse in October 2015 as Senior Director, Regulatory Affairs Gaithersburg, MD, January 6, 2016 – Vtesse, Inc....

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  7. First European site is now recruiting for VTS-270 trial in NP-C

    Great news for the NP-C community – the first European trial site for VTS-270 (2-hydroxypropyl-β-cyclodextrin) for Niemann-Pick Type C1  is now recruiting! Professor Marquardt of the Universitätsklinikum in Münster is the investigator contact for this location: CONTACT Thorsten Marquardt, Univ. Prof....

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  8. AIDNPC – teleconference minutes 26th November

    A teleconference was hosted by Orphazyme 26th November 2015, to provide an update to patient organisations regarding the development of Arimoclomol in the AIDNPC programme. These calls will continue monthly – the next scheduled to take place 14th January...

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  9. AIDNPC Clinical Programme change: Earlier initiation of Interventional study

    The INPDA and its member groups have received the following update from Orphazyme, ApS: AIDNPC Clinical Programme change: Earlier initiation of Interventional study (CT-ORZY-NPC-002; Arimoclomol treatment). After valuable dialogue with regulators and patient organizations on the design of the Observational and Interventional...

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  10. “IMAGINE”, our NP-C awareness short film, now subtitled in ten languages!

    “IMAGINE” follows the story Millie, who dreams of being a dancer yet suffers from Niemann-Pick Type C. Already viewed over 160,000 times on YouTube, we’re delighted that the English script has now been subtitled in 10 languages – Arabic,...

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