International Niemann–Pick Disease Alliance

Updates

  1. CTD Holdings Conducts Initial Review of Safety Data for its Phase I and Phase I/II Trials of Trappsol® Cyclo™ to Treat Niemann-Pick Disease Type C

    Initial data support safety and tolerability of Trappsol® Cyclo™ administered intravenously (Available for download, here) ALACHUA, FL – (Marketwired) – 03/29/2018 – CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment...

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  2. Update #18 on the Orphazyme AIDNPC Clinical Programme

    Update #18 on the AIDNPC clinical programme (arimoclomol in treatment of Niemann-Pick disease type C) Available for download in English, German, French, Spanish, Italian, and Polish.   Interim information bulletin (following the Dec. 28th call) SUMMARY 001 Study: Status...

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  3. Chin Huei Wu (巫錦輝) [NPUK: My Story]

    Chin Huei Wu, gave a rousing presentation at the NPUK Annual Family Conference & Interactive Workshop (2017) in which he gave the story of his family’s life, living with Niemann-Pick disease: Subscribe to NPUK’s YouTube channel, to never miss...

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  4. CTD Holdings Receives Rare Pediatric Disease Designation for Trappsol® Cyclo™

    ALACHUA, FL – (Marketwired) – December 6, 2017 – CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of disease with unmet medical need, today announced that it has received rare...

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  5. Orphazyme’s CEO, Anders Hinsby: Letter to NP-C Community

    Available for download in English, French, German, Italian, Polish, and Spanish   Dear Niemann-Pick type C Community, We are pleased to inform you that on November 16th Orphazyme A/S was listed on the Nasdaq OMX exchange in Copenhagen through an...

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  6. New Treatment of Disease that Swells Organs Gives New Hope to 4-year-old Girl

    Medical geneticists at the Clinical Research Center of the Hospital de Clinicas in Porto Alegre are betting on an unprecedented treatment for a rare disease, known as ASMD Niemann-Pick disease type B. The disease has no cure, and causes...

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  7. Introducing the INPDR Facebook Page!

    We are happy to introduce the International Niemann-Pick Disease Registry (INPDR)’s official Facebook page – which will provide any and all relevant updates pertaining to the INPDR.  But, what is the INPDR? The INPDR is a unifying force that has...

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  8. Update #17 on the AIDNPC Clinical Programme

    Update #17 on the AIDNPC Clinical Programme (arimoclomol in treatment of Niemann-Pick disease type C) Available for download in English, French, German, Italian, Polish & Spanish   Interim information bulletin (following the Sept. 28th call) SUMMARY 001 Study: Conclusions and...

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  9. CTD Holdings Announces Approval of Clinical Trial Application to Conduct Phase I/II Clinical Study of Trappsol(R) Cyclo(TM) in Patients with Niemann-Pick Type C Disease in Israel

    Two New Israeli Sites Now Recruiting Patients in Multicenter European Trial Full press release available for download here ALACHUA, FL – (Marketwired) – September 18, 2017 – CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage biotechnology company developing cyclodextrin-based...

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  10. Ara Parseghian: 21/5/1923 – 2/8/2017

    We are sorry to share the sad news that Ara Parseghian, passed away yesterday. Ara was a former title winning coach of Notre Dame and founded the Ara Parseghian Medical Research Foundation, which has raised over $45million for research...

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