International Niemann–Pick Disease Alliance

Updates

  1. 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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  2. 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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  3. 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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  4. 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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  5. 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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  6. 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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  7. 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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  8. 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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  9. “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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  10. Launch of dedicated website to Orphazyme’s AIDNPC clinical programme

    Copenhagen, 16 September 2015. A dedicated website to AIDNPC has been launched. The site describes Orphazyme’s clinical programme to investigate the orally available small molecule arimoclomol in Niemann-Pick disease type C. The website offers to NP-C patients a tool...

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