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    • About
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      • What is LCA?
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    Archives

    Author Archive for: "Rosanne Smyle"
    0
    By Rosanne Smyle
    In News
    Posted February 25, 2022

    Hope In Focus Statement for Connecticut’s Rare Disease Day

    Celebrating Connecticut Rare Disease Day 2022, Hope in Focus presented remarks Friday, February 25, 2022, to the Connecticut State Legislature on behalf of those living with Leber congenital [...]

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    0
    By Rosanne Smyle
    In Blog
    Posted February 25, 2022

    Rare Disease Day 2022: Hope in Focus Promotes Rare Disease Advisory Council

    Rare Disease Day 2022 kicked off today as people gathered across the country, including us here at Hope in Focus, to raise awareness and generate research for treatments and cures […]

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    0
    By Rosanne Smyle
    In Blog
    Posted February 15, 2022

    BioBonds: Innovative legislation would help fund clinical trials, including those for LCA

    A proposed Congressional Act designed to help researchers launch clinical trials for emerging treatments gives hope for getting more treatments across the finish line for people living with a [...]

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    0
    By Rosanne Smyle
    In Blog
    Posted January 28, 2022

    Let’s Chat About…ProQR’s Work in Treatments for Inherited Retinal Disease

    ProQR Therapeutics’ Founder and Chief Executive Officer shared exciting news of the deep pipeline of RNA therapies in development to treat Leber congenital amaurosis (LCA) and other [...]

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    0
    By Rosanne Smyle
    In Blog
    Posted January 17, 2022

    Let’s Chat About…Gene Therapy News for LCA13, LCA1, LCA2, and LCA4

    One of Britain’s top ophthalmologists delivered promising research news on several forms of Leber congenital amaurosis, including LCA13 (RDH12), LCA1 (GUCY2D), LCA2 (RPE65), and LCA4 (AIPL1). [...]

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    0
    By Rosanne Smyle
    In Blog
    Posted January 8, 2022

    Family Excited About New LCA5-Lebercilin Research Program

    John Mills says he would crawl over broken glass if it led to a cure for his daughter’s visual impairment caused by one of the rarest of rare inherited retinal […]

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    0
    By Rosanne Smyle
    In Blog
    Posted November 22, 2021

    Opus Genetics Launch Brings Cautious Optimism For RDH12 Families

    Allison Galloway feels cautiously excited about a new genetics company prioritizing research into her children’s form of Leber congenital amaurosis known as LCA13 (RDH12).  “I would say I’m [...]

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    0
    By Rosanne Smyle
    In News
    Posted November 9, 2021

    Hope in Focus Makes $300,000 Pledge for LCA Treatments

    The big news just keeps coming: fresh off the rebranding from Sofia Sees Hope to Hope in Focus, we are pleased to announce a pledge to raise $300,000 by 2025 […]

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    0
    By Rosanne Smyle
    In Blog
    Posted November 8, 2021

    Big Journey for Little Girl with LCA3

    Parvi Gaddam has endured a lot for a little girl genetically diagnosed at 6 months’ old with a form of Leber congenital amaurosis called LCA3 (SPATA7). Like most parents discovering […]

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    0
    By Rosanne Smyle
    In Blog
    Posted October 18, 2021

    Let’s Chat About … CRISPR and Gene Editing

    For the first time, early research data shows that a gene editing technique called CRISPR improved vision in people living with a form of Leber congenital amaurosis (LCA), according to […]

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