When Her Son Was Diagnosed with MFDM, She Couldn’t Find A Community. So She Helped Build One.
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When Her Son Was Diagnosed with MFDM, She Couldn’t Find A Community. So She Helped Build One.

As originally reported in the Cincinnati Children's, Brittney writes how she came into the world of rare diseases when her and her husband Michael decided to adopt a baby who…

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The Rare Disease Institute is Working to Bring Patients Treatments Faster
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The Rare Disease Institute is Working to Bring Patients Treatments Faster

Rare Disease Institute The Rare Disease Institute (RDI) is a new initiative aimed at improving collaboration between rare disease stakeholders. The aim is to facilitate faster diagnosis and better care…

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The Institute for Gene Therapies is Working to Improve Policy and Access for Charcot-Marie-Tooth Disease and Others
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The Institute for Gene Therapies is Working to Improve Policy and Access for Charcot-Marie-Tooth Disease and Others

The Institute for Gene Therapies (IGT) is a new development, aimed at improving the speed of development and accessibility of gene therapies. Gene Therapy Many rare diseases are caused by…

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A First for CRISPR: Editing a Gene in the Retinal Cells of a Leber Congenital Amaurosis Patient

  NPR News recently featured an article announcing that scientists at Portland’s Casey Eye Institute have attempted to use CRISPR to edit a gene with the DNA still in place.…

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