International Niemann–Pick Disease Alliance

News

  1. Orphazyme: Update #13 on the AIDNPC Clinical Programme

    Update #13 on the AIDNPC clinical programme (arimoclomol in treatment of Niemann-Pick disease type C)  (Available for download in English, French, German, Italian, Polish, and Spanish) Conference call with patient organizations (26 JANUARY ’17)   SUMMARY 002 Study: Recruitment...

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  2. Clinical trial extended to Australia in bid to combat Niemann-Pick Disease

    The following article has been adapted from stories in the Herald Sun and Bendigo Advertiser: Two siblings from the state of Victoria, Australia, have became the first in the country to take part in a clinical trial for an experimental drug...

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  3. CTD Holdings Receives FDA Fast Track Designation for Development of Trappsol® Cyclo™ to Treat Niemann-Pick Disease Type C

    ALACHUA, FL — (Marketwired) – 1/17/17 — CTD Holdings, Inc. (OTCQB: CTDH), a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track...

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  4. Wylder Nation link with Perlara

    The Wylder Nation Foundation, which seeks treatment for acid sphingomyelinase deficiency (ASMD) NPD type A, has partnered with biotech company Perlara: Perlara PBC, a company with a focus on ‘finding treatments for rare diseases previously believed to be too...

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  5. Perlara PBC Announces Patient Advocacy Group Partnerships

    Perlara PBC Announces Patient Advocacy Group Partnerships – News Press Release | PharmiWeb.com SAN FRANCISCO, Jan. 10, 2017 /PRNewswire/ — Perlara PBC, a global patient portal and drug discovery platform company for the 1 in 10 families affected by...

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  6. Vtesse Announces Dosing of First Patient in Australia in Phase 2b/3 Clinical Trial of VTS-270 in Niemann-Pick Type C1 Disease

    – Clinicians Continuing to Enroll Children with NPC at Trial Sites in the United States, Germany, the United Kingdom, France, Spain, and Turkey    GAITHERSBURG, Md., Jan. 10, 2017 /PRNewswire/ — Vtesse, Inc., a company committed to developing medicines to benefit...

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  7. ASMD NP-B Enzyme Therapy: Luiz Henrique

    Just last week we happily reported that three year old Isadora, had became the first patient of her age to receive enzyme therapy for ASMD Niemann-Pick disease type B. This tremendous news filled everyone connected to the INPDA with a sense...

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  8. Vtesse December 2016 Newsletter: Update on the Clinical Trial of VTS-270: The NPC Study

    Update on the Clinical Trial of VTS-270: The NPC Study December 2016 Newsletter (Available for download in Dutch, English, German, Latin American Spanish, Mandarin, Persian, and Spanish) Update on the Vtesse Clinical Trial of VTS-270: Currently, we have enrolled 43...

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  9. Orphazyme: Update #12 on the AIDNPC Clinical Programme

    Update #12 on the AIDNPC clinical programme (arimoclomol in treatment of Niemann-Pick disease type C)  (Available for download in English, French, German, Italian, Polish, Spanish) Conference call with patient organizations (22 DECEMBER ’16) SUMMARY 002 Study: Recruitment into the...

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  10. The First Three Year Old Patient to Receive Enzyme Therapy for Niemann-Pick Type B

    A constant smile and strong personality are just two traits of Isadora, a Brazilian girl who has became the first three year old in the world to receive enzyme therapy for Niemann-Pick type B disease. Upon hearing this news...

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