Usher syndrome is the most common genetic cause of combined deafness and blindness. More than 400,000 people are affected by this disorder worldwide. There is currently no cure for Usher syndrome. 

You have the power to change that.

The Usher Syndrome Coalition is working to raise awareness and accelerate research, while providing information and support to impacted individuals and families. We strive to be the most comprehensive resource for the Usher syndrome community, bridging the gap between researchers and families. Learn more and get involved.  

The USH2019 Early Bird Rate has been extended to March 15th and there are new opportunities to connect at this annual Usher syndrome event!

The Sanford Health Lorraine Cross Award worth $1 million was established to award game-changers in medicine. The award is not to have people to live forever, but to “live life without suffering.” The winners of the award are Dr. Jean Bennett and Dr. Katherine High of the University of Pennsylvania, pioneers of gene therapy research. The award is in recognition for the improvements in gene therapy that lead to an FDA- approved treatment for Leber’s congenital amaurosis.

What this means for Usher syndrome: This award not only reflects the importance of gene therapy for the treatment of genetic disorders but could accelerate research in Usher syndrome through gene therapy.

The United States Patent & Trademark Office (USPTO) has approved the usage of mesencephalic-astrocyte-derived neurotrophic factor (MANF) or cerebral dopamine neurotropic factor (CDNF) as a treatment for various retinal disorders including retinitis pigmentosa, macular degeneration, or glaucoma. Both factors can be administered as an eye drop or by intravitreal injection. MANF is believed to have potential because it is a naturally-occurring protein produced by the body to reduce or prevent cell death in response to injury or disease through unfolded protein response.

What this means for Usher syndrome: Since MANF reduces or prevents cell death, in the case of Usher syndrome, it could prevent photoreceptor cells from dying, and thus preserve vision.

Join us for our 11th annual USH Connections Conference in Philadelphia, Pennsylvania on July 13, 2019. Attendees describe this conference as "life-changing," an event with a positive atmosphere and a genuine sense of community. At the USH Connections Conference, you will connect face-to-face with hundreds of people who "get it", meet and network with others facing the same challenges, and learn the latest on developing treatments from leading USH researchers.

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