As we reflect on the past year, we’re filled with gratitude for the curiosity, compassion, and generosity that fuel our USH community. Every discovery we share and every story we tell is made possible by readers like you, people who believe in the power of science to illuminate, connect, and inspire.
Your support keeps Grounded in Science thriving. From groundbreaking research updates to powerful community voices, it's your generosity that drives the work moving us forward.
As Giving Tuesday approaches, we invite you to make a donation and help continue this mission. Every contribution, large or small, amplifies the science and stories that bring hope and understanding to those living with Usher syndrome. |
|
|
RESEARCH SPOTLIGHT
New Hope for Vision: Nanoscope Therapeutics’ MCO-010 Moves Closer to FDA Approval | |
|
Back in October 2024, Nanoscope Therapeutics announced plans to begin a rolling submission to the FDA for its potential treatment, MCO-010, following a positive meeting with the agency. Less than a year later, that plan is becoming a reality. In July 2025, Nanoscope officially started its Biologics License Application (BLA) for MCO-010, a gene-agnostic optogenetic therapy designed to help people with retinitis pigmentosa (RP), including those with Usher syndrome, regain some ability to see light and shapes.
Over the last few months, Nanoscope has issued multiple press releases highlighting recent study results showing lasting benefits. In the REMAIN study, people with advanced RP maintained meaningful vision improvements for three years after a single MCO-010 injection, with no serious side effects. A separate five-year safety study confirmed the treatment remained safe and stable over time. These findings suggest that MCO-010 could improve long-term vision for people who currently have no treatment options.
MCO-010 is also showing promise beyond RP. In the STARLIGHT study, people with Stargardt disease, another inherited retinal condition, experienced measurable vision improvements and no major safety issues. Because MCO-010 works without targeting a specific gene, it may one day help a wide range of retinal conditions, including Usher syndrome.
What this means for the Usher syndrome community: For people and families affected by Usher syndrome, these results bring hope. Since MCO-010 doesn’t rely on fixing one specific gene, it could one day help those with different or unknown mutations who have never had treatment options. While the FDA review continues, Nanoscope’s continued progress marks a major step toward restoring sight and possibly independence for those living with both hearing and vision loss.
Link to article
Nanoscope Therapeutics newsroom |
|
|
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 | |
|
October 23, 2024: PCDH15 Dual-AAV Gene Therapy for Deafness and Blindness in Usher Syndrome Type 1F Models
Gene therapies are a promising method to treat all forms of Usher syndrome. The goal is to deliver a healthy copy of a gene - or tools to repair it - into cells, usually using a harmless virus called AAV (adeno-associated virus). But many Usher genes are very large, and AAV has a strict size limit on the amount of DNA it can carry. This means most Usher genes do not fit into a single AAV.
To address this problem in USH1F, which is caused by mutations in the PCDH15 gene, researchers tested a “dual-AAV” strategy. They split the PCDH15 gene into two smaller segments, each packaged into its own AAV. When cells were given both AAVs, the two pieces rejoined within the cell to form a full-length, functional PCDH15 protein. The results were impressive: in mice missing normal PCDH15 protein, this therapy restored both hearing and balance. The approach also worked in human retinal organoids (lab-grown eye tissues that mimic the human retina) and even in the retina of a monkey, showing the protein was correctly produced in different models.
What this means for the Usher syndrome community: This “dual-AAV method” could be used for any large USH gene that doesn’t fit into one AAV. Other researchers are already testing this approach for USH1B (MYO7A), another very large gene. As more studies show success, this strategy could expand to more USH genes, bringing hope for future treatments that could work across many subtypes of the disease. Read the article. |
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: USH Connection and Community Stories |
|
|
As we approach the holiday season, we want to take a moment to say thank you to everyone who has shared their story with us. We’ve loved reading your stories of connection, strength, and resilience that truly capture the heart of the Usher syndrome community.
We’re excited to highlight one of these incredible stories today and will continue sharing more throughout the holidays. Each story reminds us how powerful our community is when we come together.
Among the many stories shared, one that beautifully reflects the strength of our community comes from Katie Fromholt in Kentucky, whose son lives with Usher syndrome:
“The Usher Syndrome Coalition has been a source of connection, understanding, and community for our family. When you first get the diagnosis, it can feel isolating and overwhelming.
Thirteen years ago, we were connected with Mark Dunning and shown what the (still in its early days) Coalition could do to support us. We found it a place where people already understood. It’s helped us see that we’re part of something bigger, and that there’s a network of people working every day to support, inform, and bring hope to families like ours.
For our son, it’s meant seeing role models who show that having Usher syndrome doesn’t define or limit who you can become. We have hope in our son’s resilience and determination. What we wish others understood is that living with Usher syndrome isn’t just about loss. It is about adaptation, strength, and connection as well.
It’s hard to pick just an event or time, because there have been so many moments that stand out. But what stays with me most are the countless connections we’ve made, both online and in person, that have made this journey feel less lonely. Whether it’s a message from another parent across the country or a hug from a family we finally meet in person, those connections remind us that we’re part of a community that lifts each other up every step of the way."
If you haven’t yet shared your experience, there’s still time! Our submissions are still open as we collect stories to feature in the new year. |
Whether you’ve found connection, support, or hope through the Usher Syndrome Coalition, your story matters. By sharing it, you can inspire others, especially those newly diagnosed or just finding the Coalition, and help them know they’re not alone. |
|
|
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.
|
|
|
SignUp – Sign Language for Netflix & Disney+ is a free Chrome extension that overlays sign-language interpretation videos (in ASL, BSL, or ISL) onto shows and movies on Netflix and Disney+. It’s designed to be simple and easy to use, making entertainment more accessible and helpful for anyone learning sign language.
Maddie + Triggs is a warm, musical-adventure animated series for young children and their families, that celebrates difference, sensory discovery and the power of sound, while also being inclusive for low vision audiences. You can listen to the show on the RTÉ website through their podcast player.
Send your USH Tips to info@usher-syndrome.org |
|
|
Our Contact Information
*{{Organization Name}}*
*{{Organization Address}}*
*{{Organization Phone}}*
*{{Organization Website}}*
*{{Unsubscribe}}* |
| |
|
|
