The latest USH blog posts and various news items impacting the Usher syndrome community.
Living with Usher syndrome presents daily challenges, but as Molly Watt shares in this patient perspective featured by MedJournal 360, it also fosters remarkable resilience.
As government and NIH funding decisions unfold, we remain committed to advocating for continued progress in research efforts that are critical to our community.
Living with Usher syndrome presents daily challenges and requires constant adaptation as we navigate our unique journeys through deafblindness. However, this diagnosis should never define us or limit our potential.
Nacuity Pharmaceuticals, Inc. announced that its investigative therapy, NPI-001, for treating retinitis pigmentosa (RP), has received both Fast Track Designation and Orphan Drug Designation from the U.S. FDA.
As we welcome the new year, many people turn to resolutions. Let’s consider an alternative: setting intentions.
Registration is open for USH2025, the 5th International Symposium on Usher Syndrome! The Scientific Conference and Congrestival, hosted by the Dutch Usher Syndroom Foundation, will take place in Nijmegen, the Netherlands, on June 19, 20 and 21, 2025.
Learn about the Usher Syndrome Coalition’s partnership with Sepul Bio to support enrollment in the LUNA clinical trial, a Phase 2b study testing ultevursen, a potential RNA therapy for USH2A-mediated retinitis pigmentosa.
Sepul Bio launches the LUNA Phase 2b clinical study to test ultevursen, a potential mRNA therapy aimed to help people with Usher syndrome type 2a maintain their vision.
The holiday season is a time of joy and togetherness, but it can also bring stress and overwhelm. It’s easy to put yourself last in the hustle of shopping, planning, and attending events...
As we approach the season of giving thanks, it can be helpful to remember that gratitude and kindness are simple practices that can have a positive impact on how we feel.
Personal health literacy is defined as the degree to which individuals can find, understand, and use information and services to inform health-related decisions and actions for themselves and others.
In late 2024, AAVantgarde shared exciting announcements on the first participant dosage, Orphan Drug Designation, and early safety data in the Phase 1/2 LUCE-1 clinical trial for vision loss caused by Usher syndrome type 1B.
Exploring the challenges of living with Usher syndrome and the importance of disability equity in the workplace.
BlueRock Therapeutics LP has announced that the U.S. Food and Drug Administration (FDA) has cleared OpCT-001, its investigational product, an induced pluripotent stem cell (iPSC)- derived cell therapy, through an Investigational New Drug (IND) application.
Attending the Usher Syndrome Coalition's USH Connections Conference for the first time can evoke a wide range of emotions. It can be incredibly empowering to connect with others who truly understand your experience, who "get it." At the same time, it may raise questions about the future, such as, "What will my life be like?" or "What can I do now if there aren’t yet any available treatments?"
Founded in 1995, DeafLEAD provides extensive support to Deaf, Hard of Hearing, Late-Deafened, and DeafBlind individuals.
This story highlights a young Korean boy who uses his pseudonym Yeong-ho. Yeong-ho was diagnosed with USH2A at Seoul National University Hospital as a newborn.
This article highlights a new partnership between GenKOre and Revvity.
USH1F is caused by mutations in the protein Protocadherin-15 (PCDH15). Because PCDH15 is a very large protein, the genetic sequence that cells use to make PCDH15 protein is too long to fit into the viruses that are used to deliver gene therapy treatments.
To develop successful treatments, scientists must understand disease etiology and its corresponding biomarkers, which are biological molecules.
The benefits of movement are many: movement helps you stay healthy, improves your mental health, and builds community and self-esteem. Here are some tips to make movement and exercise more “Usher syndrome friendly.”
Italy’s Center for Rare Ocular Diseases at the University of Campania designed and conducted a longitudinal natural history study of European patients with confirmed Usher syndrome diagnosis and biallelic MYO7A variants.
Eye doctors use various tools to determine whether cone photoreceptor cells are working. In this study, researchers compared these tools to a newer, more detailed imaging method called adaptive optics (AO).
Inherited retinal diseases (IRDs) are complex eye conditions caused by genetic mutations that lead to progressive vision loss.
As a mental health provider, I understand that multiple feelings can be true at the same time. It is possible to both value and uphold the ability to self-advocate while sometimes needing a break.
