Research Articles - Usher Syndrome
Research to save and restore vision for people with Usher syndrome is receiving a significant boost, thanks to a three-year, $450,000 grant awarded to the Foundation Fighting Blindness by the Marjorie C. Adams Charitable Trust. The funds will be used to support the Foundation’s diverse portfolio of Usher syndrome-related projects, including emerging gene therapies, stem-cell treatments and pharmaceuticals.
- A few weeks ago, 15 of the world’s leading retinal researchers, all funded by FFB’s Translational Research Acceleration Program (TRAP), gathered for an annual meeting in Las Vegas to discuss their progress in developing vision-saving treatments. Most are poised to move into clinical trials within the next two to five years.“It’s an exciting time in translational research for retinal diseases,” says Patricia Zilliox, Ph.D., chief drug development officer at the
- November 17, 2014 - The Foundation Fighting Blindness (FFB) recently updated its website by adding Spanish language translations of some of its resource materials to better serve patients and others interested in learning about retinal diseases.Those wishing to view the materials in Spanish may click on the link En Espanol in the resources section of the Foundation’s website.
Lilly Diuble, an 11-year-old who’s helped raise more than $100,000 for the Foundation through her family’s VisionWalk teams, was featured last night in the “Making a Difference” segment of NBC Nightly News with Brian Williams. Lilly lives with her family in Manchester, Michigan, which was visited by NBC’s Peter Alexander and a production crew last week.
The Foundation Fighting Blindness recently awarded $125,000 to the Centre Hospitalier National d’Ophtalmologie des Quinze-Vingts, a clinical trial site for UshStat®, a gene therapy developed by Oxford BioMedica for people with Usher syndrome type 1B (USH1B). The funding level is the amount needed for the site, located in Paris, France, to take part in the study.
The Phase I/II, 18-person study began at Oregon Health & Science University
Dr. Muna Naash, a Foundation-funded researcher from the University of Oklahoma Health Sciences Center, used a nanoparticle-based gene therapy to provide long-term preservation of vision in mice with Stargardt disease. A study recently published online in the Journal of Clinical Investigation shows that the innovative treatment approach has the potential to be an advantageous alternative for delivering therapeutic genes to
Oxford BioMedica, a leading gene therapy development company in the United Kingdom, has received a positive interim safety review of its ongoing Phase I/IIa clinical trial for the UshStat® gene therapy for people with Usher syndrome type 1B. The first three patients experienced no adverse events for up to six months after treatment. The positive review by the study’s Data Safety Monitoring
Oxford BioMedica, a leading gene therapy development company in the United Kingdom, announced today that it has voluntarily paused participant recruitment for its retinal gene therapy clinical trials due to the detection of very low concentrations of potential impurities in material used to make the treatments. The clinical trials impacted by this action are for
- A 75-year-old woman with virtually complete vision and hearing loss from Usher syndrome went to the ophthalmology department at the Hospital Clinico San Carlos in Madrid, Spain, a while back, reporting some curious symptoms. At night she was hearing cabaret music and seeing what the doctors called “complex visual hallucinations.”
After a thorough neurological examination, the doctors determined that she was “aware of her reality” and wasn’t suffering from any mental illness or