International Niemann–Pick Disease Alliance

Updates

  1. Sanofi Genzyme: Clinical trial update regarding ASCEND Trial for adults with ASMD

    Following an update meeting with Genzyme regarding the Phase 2/3 Olipudase Alfa trial in adult patients (18+ years old) with ASMD (Niemann Pick Disease Type B), we are pleased to provide an update on the status of the Sanofi...

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  2. CTD Holdings Ships Additional Trappsol® Cyclo™ to Brazil

    CTD Holdings Ships Additional Trappsol® Cyclo™ to Brazil Trappsol® Cyclo™ Is the Exclusive Form of Hydroxypropyl Beta Cyclodextrin Purchased by the Government of Brazil for the Compassionate Treatment of Niemann-Pick Disease Type C Available for download, here ALACHUA, FL...

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  3. CTD Holdings Enrolls First Patient in European Phase I/II Clinical Trial of Trappsol® Cyclo™ for Treatment of Niemann-Pick Disease Type C

    CTD Holdings Enrolls First Patient in European Phase I/II Clinical Trial of Trappsol® Cyclo™ for Treatment of Niemann-Pick Disease Type C Company Expects Final Data from Trial by End of 2018 Alachua, FL – (Marketwired) – June 21, 2017...

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  4. The Annual VML Members’ Weekend (26th-28th May 2017)

    The Annual VML Members’ Weekend was held from Friday 26 May to Sunday 28 May 2017 at the Domaine de Chalès in Nouan-le-Fuzelier (41) France – as ever it was an occasion filled with fun as the community came...

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  5. CTD Holdings Announces Appointments of Two Patient Liaisons to Enhance Outreach to Niemann-Pick Disease Type C Families and Caregivers

    CTD Holdings Announces Appointments of Two Patient Liaisons to Enhance Outreach to Niemann-Pick Disease Type C Families and Caregivers Liaisons to Provide Counsel to Patient Families Around Recently Initiated Phase I and Phase I/II Clinical Trials ALACHUA, FL –...

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  6. Pactimo Online Charity Shop – Now Open!

    The Pactimo online charity shop, which seeks to support the International Niemann-Pick Disease Alliance (INPDA), is now open! Summer is almost in full swing so why not have a look at our biking suits or sports T-shirts for either...

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  7. Orphazyme: Update #16 on the AIDNPC Clinical Programme

    (Available for Download in English, German, French, Spanish, Italian, and Polish) Interim Information Bulletin SUMMARY 002 Study: All patients have now started treatment with study medicine Orphazyme to present at Spanish NPC Association on June 24th     The...

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  8. The 21st Niemann-Pick Sielbstilfegruppe Family Support Meeting – Overview

    The 21st  Niemann-Pick Sielbstilfegruppe family support meeting took place in Kassel last weekend on Saturday 27th May.  This was the 17th year that it has been held in the same location, one ideally positioned in the geographical centre of...

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  9. CTD Holdings to Host Webinar to Provide Update on Trappsol® Cyclo™ Drug Development Program for Treatment of Niemann-Pick Disease Type C

    Download Here Opportunity for Patients, Families, and Caregivers to Hear Additional Details on Recently Initiated Phase I and Phase I/II Clinical Trials ALACHUA, FL — (Marketwired — May 16, 2017) — CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage...

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  10. Orphazyme Completes Its Enrolment of Patients for Phase 3 Clinical Trial in Niemann-Pick Type C Disease

    ORPHAZYME COMPLETES ENROLMENT OF PATIENTS FOR PHASE 3 CLINICAL TRIAL IN NIEMANN-PICK TYPE C DISEASE Copenhagen, 11 May 2017 Download Here Orphazyme ApS, a Danish biotech company developing novel therapies for the treatment of protein misfolding diseases, today announced...

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