Grounded in Science
A balance of research news and well-being for the Usher syndrome community.
Each year on March 3, World Hearing Day, led by the World Health Organization, shines a light on the importance of hearing health around the world. For the USH community, this day is deeply personal. Usher syndrome is the leading genetic cause of combined hearing and vision loss. People with Usher syndrome are born with or develop hearing loss later in life.
Grounded in science and driven by hope, research has helped doctors better understand the genes that cause Usher syndrome and improve early diagnosis through newborn hearing screening and genetic testing. Early identification opens the door to hearing technology, vision care, educational support, and connection to community.
We are committed to awareness, research, and collaboration - working together to ensure that no one with Usher syndrome feels alone and that progress toward treatments continue.
RESEARCH SPOTLIGHT: MCO-010 Three-Year Data Shows Lasting Vision Improvements
In our November 2025 issue, we shared that Nanoscope Therapeutics had started the process of applying for FDA approval for MCO-010, a potential treatment for retinitis pigmentosa (RP) that could help people with Usher syndrome. This month, we have a meaningful update to share.
At the end of February 2026, researchers presented three-year results from two clinical studies, RESTORE and REMAIN, at the Macula Society Annual Meeting. These results are an important part of the ongoing FDA approval process.
What is MCO-010? MCO-010 is a gene therapy delivered as a single injection into the eye. It works by activating a different layer of retinal cells to respond to light, bypassing the cells that have already been lost to RP. Because it does not target one specific gene mutation, it may be able to help people regardless of which gene caused their vision loss, including across different Usher syndrome subtypes.
What did the new data show? Across both studies, people with advanced RP who received MCO-010 showed meaningful improvements in visual acuity, roughly the equivalent of reading three additional lines on an eye chart. Those gains lasted through three years of follow-up, and no treatment-related serious side effects were reported.
What does this mean for the Usher syndrome community? Vision loss due to RP affects people with all types of Usher syndrome. Because MCO-010 does not rely on correcting one specific gene, it could one day be an option for people across our community, including those with less common subtypes or mutations that do not yet have a dedicated treatment in development. Nanoscope's rolling BLA submission to the FDA is currently underway. If approved, MCO-010 would be a one-time injection that does not require genetic testing to qualify.
To stay informed about clinical trials that may be relevant to you, join the USH Trust.
Join the USH Trust to stay updated on clinical trials and opportunities to participate. Only ten questions need to be answered to register.
In Case You Missed It: Science News Feature
Tiny Lab-Grown Organs Are Helping Scientists Study and Treat Usher Syndrome
April 8, 2025: Scientists at Boston Children's Hospital and Harvard Medical School are studying how the body grows and repairs itself by creating tiny "mini-organs" in the lab. These mini-organs, called organoids, are made from human stem cells. Some are designed to resemble parts of the inner ear, while others resemble skin. Inner ear organoids can grow the special hair-like cells needed for hearing, and skin organoids can form layers of skin and even tiny hairs.
By watching these mini-organs grow, researchers can observe how genetic diseases affect the body in real time. For people with Usher syndrome, organoids made from a patient's own cells can reveal how hearing cells begin to stop working due to specific gene mutations. This gives scientists a way to test new treatments early and safely, without waiting for animal or human trials. In some studies, skin organoids were placed onto mice and grew real human hair, showing that lab-grown tissue can survive and function in the body.
This same research team is actively using these tools to study Usher syndrome specifically, including testing treatments that aim to repair the damaged hair cells responsible for hearing loss.
What this means for the Usher syndrome community: Organoids give researchers a faster, more human-relevant way to understand how Usher syndrome affects the inner ear and to test new therapies before they reach clinical trials. Each advance in this technology brings scientists one step closer to treatments that target the root cause of hearing and vision loss in our community.
DISCLAIMER: The Usher Syndrome Coalition does not provide medical advice nor promote treatment methods. USH Science News is intended to help summarize more complex literature for the community to use at their own discretion.
For more science news, check out our Science News page, organized by treatment approach and type of Usher syndrome.
On Well-Being: What if disability isn’t something to fix?
Research updates like the one above remind us how far science has come. But progress doesn't only happen in a lab. Rebecca Alexander, accepting the Helen Keller Achievement Award from the American Foundation for the Blind, offered a reminder that how we live with Usher syndrome matters just as much as what we discover about it.
Recently posted by Rebecca Alexander on Instagram, these words are timely as we recently observed Rare Disease Day and World Hearing Day. The message is simple, powerful, and transcribed here:
"My purpose, my legacy
is not only to educate and inform
but also to show people what having a disability looks like
My legacy is to eradicate the stereotypes
and assumptions people often make about what it means to have a disability
in hopes that it will elicit curiosity, understanding
and a greater sense of community."
The caption reads: “What if disability isn’t something to fix, but a different way of being human? As a deafblind woman, my purpose on stage isn’t just to educate or inspire. It’s to challenge the idea that a cure is the goal and to ask what becomes possible when we invest in access, inclusion, and thoughtful design instead."
Video Description: Rebecca stands at a podium as she accepts the Helen Keller Achievement award from the American Foundation for the Blind. She is wearing a black formal dress with her hair pulled back in a low bun.
Disclaimer: The information and resources on this website are provided for educational and informational purposes only and do not provide medical or treatment advice. Check out our mental health resources page on our website.
USH Tip
USH Tip: How to Transcribe a Video for Accessibility
Use a Website: Copy the video link and paste it into a site like RecCloud or Kapwing. These sites can turn the video sound into text automatically.
Download and Upload: Save the video to your computer or phone. Then, upload it to a tool like Otter.ai or HappyScribe to get a typed-out version.
Check for Captions: On your phone, tap the three dots on the video. Sometimes there is a 'Show Transcript' button you can click to see the words.
Use Live Captions: You can turn on 'Live Captions' in your phone's settings. This will show the words on the screen while the video plays.
Send your USH Tips to info@usher-syndrome.org
USHER SYNDROME DATA COLLECTION PROGRAM
As the world continues to get to know the individuals living with Usher syndrome, it's a great time to join the Usher Syndrome Data Collection Program - the USH DCP - so researchers can better understand this diagnosis.
If you'd like additional support enrolling, please reach out to Yael Saperstein, our Community Enrollment Coordinator for the USH DCP. Yael is an expert on the enrollment process, accessibility, and guiding new participants every step of the way. Contact Yael here: y.saperstein@usher-syndrome.org.
