International Niemann–Pick Disease Alliance

Updates

  1. International Niemann Pick Disease Alliance – 6th Biennial “Face to Face” Meeting

    International Niemann Pick Disease Alliance 6th Biennial “Face to Face” Meeting 14-16 June 2019   Radisson Blu Hotel Lyon 129 Rue Servient, 69003 Lyon, France https://www.radissonblu.com/en/hotel-lyon   We are pleased to announce that the 6th Biennial INPDA Face-to-Face Meeting...

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  2. CTD Announces New Expanded Access Program in Taiwan using Trappsol® Cyclo™ for Niemann-Pick Disease type C

    Program is open to two patients who completed CTD’s clinical trial with Trappsol® Cyclo™ in the United States ALACHUA, FL – (Accesswire) – May 07, 2019 – CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage...

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  3. Raphaël Beaugillet: Road Para-Cycling World Cup

    My name is Raphaël Beaugillet, I come from France and I am a friend of Alix (age 36) and Zita (age 30) both of whom are affected by the ultra-rare Niemann-Pick-C disease (NP-C). I will participate in the Road...

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  4. CTD Holdings Announces Plan to Launch Clinical Trial of Trappsol® Cyclo™ in Alzheimer’s Disease

    ALACHUA, FL – (Globe Newswire) – March 14, 2019 – CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of disease with unmet medical need, announced today that the company has...

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  5. IntraBio Clinical Trial Authorization Application Approved by the MHRA for the Treatment of Niemann-Pick Disease type C (NPC)

    OXFORD, UK, March 11, 2019– IntraBio Inc., a late-stage biopharmaceutical company, today announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved its Clinical Trial Authorisation (CTA) Application for Clinical Trial IB1001-201 with its lead compound...

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  6. CTD Holdings Announces Grant to U.S. Non-Profit Organization to Support Patient Participation in Informational Meetings with Regulators

    CTD Holdings Announces Grant to U.S. Non-Profit Organization to Support Patient Participation in Informational Meetings with Regulators Support is part of CTD’s efforts to recognize Rare Disease Day 2019 ALACHUA, FL – (Globe Newswire) – February 28, 2019 –...

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  7. JJ’s Story: Living with ASMD

    JJ was born with an enlarged spleen and liver and it wasn’t until the age of three that he was diagnosed with Acid Sphingomyelinase Deficiency (ASMD), also known as Niemann-Pick disease type B. In the following video he describes...

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  8. Orphazyme reports positive results from full data set of Phase II/III arimoclomol trial in Niemann-Pick disease Type C (NPC)

    Orphazyme A/S Company announcement                                                                                                                    No. 01/2019                                                                                                                                         Company Registration No. 32266355  Primary endpoint shows 74% reduction in disease progression after 12 months compared to placebo control (p-value =0.0506) Biomarker results demonstrate statistically significant biological response to treatment Orphazyme to engage...

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  9. CTD Announces Partnership with United BioSource LLC to support the Extension Protocol for its Phase I Clinical Trial in the United States

    CTD Announces Partnership with United BioSource LLC to support the Extension Protocol for its Phase I Clinical Trial in the United States Collaboration will allow patients in Extension Protocol to receive CTD’s drug intravenously at home ALACHUA, FL –...

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  10. CTD Announces Presentation at Upcoming Conference on Lysosomal Storage Diseases

    Annual conference offers opportunities for academic researchers and industry representatives to exchange information on novel therapeutic interventions. ALACHUA, FL – (Globe Newswire) – January 09, 2019 – CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops...

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