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    The MLD Foundation Supporting Drug Development - 10 month(s) ago

    Dean Suhr of the MLD Foundation explains their philosophy when it comes to supporting research for metachromatic leukodystrophy (MLD).

    Source: CheckRare
    Categories: Hem/Onc News and Journals, Latest Headlines
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      Dean Suhr of the MLD Foundation provides an overview of the philosophy of their foundation when it comes to supporting research for metachromatic leukodystrophy #MLD #checkrare #rarediseses @MLDfoundation https://t.co/s35S8AxRwn

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    Clinical Trial Development Challenges - 10 month(s) ago

    Rennie McCarthy, CEO at Stealth Biotherapeutics, discusses challenges in developing clinical trials for ultra-rare diseases.

    Source: CheckRare
    Categories: Hem/Onc News and Journals, Latest Headlines
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      Rennie McCarthy, Chief Executive Officer at Stealth Biotherapeutics, discusses clinical trial development challenges for ultra-rare diseases #checkrare #rarediseases @rarediseasesnet https://t.co/rq19VCjANO

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    Basic Mechanisms and Model Systems in Lysosomal Disorders - 10 month(s) ago

    Learn how models are used to test treatment efficacy and how they’re used in Niemann-Pick type C and Gaucher disease.

    Source: CheckRare
    Categories: Hem/Onc News and Journals, Latest Headlines
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      CME: Mia Horowitz, Ph.D., at @TelAvivUni, Ricardo Feldman, Ph.D., @UofMaryland and Andrés D. Klein, Ph.D., at @ICIMUDD, Chile, discuss different model systems used in #Gaucherdisease and how models are used to test treatment efficacy. https://t.co/Jgtockuxvw https://t.co/M49Wrq58mB

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    FDA Approves First Gene Therapy for Hemophilia A - 10 month(s) ago

    The price for this one time infusion will be $2.9 million.

    Source: CheckRare
    Categories: Latest Headlines, Rare Disease
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      The U.S. Food and Drug Administration #FDA has approved BioMarin’s gene therapy [valoctocogene roxaparvovec-rvox (Roctavian)] to treat adults with severe hemophilia A #checkrare #hemophiliaA @hemophiliafed @NHF_Hemophilia https://t.co/EbyRYICHlS

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    Myotonic Dystrophy Type 1 Management - 10 month(s) ago

    Dr. Nicholas Johnson, of Virginia Commonwealth University, explains myotonic dystrophy type 1 and how it is currently managed.

    Source: CheckRare
    Categories: Hem/Onc News and Journals, Latest Headlines
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      Nicholas Johnson, MD, Associate Professor in the Department of Neurology at Virginia Commonwealth University, explains myotonic dystrophy type 1 and how it is currently managed #checkrare #rarediseases #DM1 @MyotonicStrong @mdsguk https://t.co/4HJXrQlNZn

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    Fighting the Rare: Lafora Disease Documentary - 10 month(s) ago

    Dr. Jordi Duran and parent Niki Markou discuss Fighting the Rare, a documentary focused on Lafora disease.

    Source: CheckRare
    Categories: Hem/Onc News and Journals, Latest Headlines
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      Jordi Duran, PhD, Associate Professor at Institut Químic de Sarrià in Barcelona, and Niki Markou, parent of a child with Lafora disease, discuss the documentary Fighting the Rare #checkrare #rarediseases #FightingtheRare @Chelseashopela1 @FightingTheRare https://t.co/D7ExcHWK8M

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