Grounded in Science
A balance of research news and well-being for the Usher syndrome community.
Save the Date: USH2027 International Symposium on Usher Syndrome
David Rubenstein Treehouse, Harvard University, Boston, Massachusetts
Scientific Symposium: Thursday and Friday, October 7-8, 2027
Community Day: Saturday, October 9, 2027
The International Symposium on Usher Syndrome is coming to Boston! This event brings together scientists, clinicians, and the Usher syndrome community from around the world.
For the first two days, researchers and clinicians will share their latest work, talk through shared challenges, and build new collaborations.
On Saturday, the symposium opens to the whole community. People with Usher syndrome, their families, and researchers will come together to learn about the latest science, learn from each other, and talk about what comes next.
We'll share more details, including registration and accessibility information, in the coming months. Mark your calendar now!
USH2027 is co-hosted by the Usher Syndrome Coalition and the Usher Syndrome Society, together with Boston Children’s Hospital, in collaboration with the Usher 1F Collaborative, LSU Health Sciences Center New Orleans, Massachusetts Eye and Ear, Radboud University Medical Center, Moorfields Eye Hospital and University College London.
RESEARCH SPOTLIGHT: Nacuity Pharmaceuticals
October 28, 2026: Nacuity Pharmaceuticals announced late last month that it dosed the first patient in its Phase 3 study, SLO-RP Preserve. It’s the company’s first Phase 3 trial, the last major stage before seeking FDA approval. The drug, NPI-001, is a twice-daily pill whose active ingredient is N-acetylcysteine amide, or NACA. NPI-001 already holds three special FDA designations, Fast Track, Breakthrough Therapy, and Orphan Drug status, a status reserved for rare disease treatments. All three are meant to speed up its development and review.
NPI-001 works by boosting glutathione, a natural antioxidant made by the body. Antioxidants protect cells from oxidative damage, a kind of wear and tear caused by unstable molecules inside cells. Researchers believe this damage contributes to retinitis pigmentosa (RP) by harming the retina’s light-sensing cells over time. The hope is that more glutathione can protect those cells and slow the vision loss that RP causes.
The trial will study up to 80 adults with RP caused by Usher syndrome over 24 months. Patients will get either NPI-001 or a placebo, a pill with no active drug. Neither patients nor doctors will know who is getting which. This setup is called double-masked, and it helps keep results unbiased. The study is enrolling at sites in the United States and Australia. The first patient was dosed in Dallas, Texas.
What this means for the Usher syndrome community: There is currently no approved treatment for this form of RP. Because NPI-001 targets cell damage rather than a specific gene, it could help people with Usher syndrome caused by different genes. The study will test how well the drug works and gather more information about its safety. While the results are still ahead, reaching Phase 3 is an important step toward a possible treatment.
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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
Nanoscope’s MOGENRY Gets Priority Review in Japan for Inherited Retinal Diseases
October 1, 2026: Nanoscope Therapeutics announced a big step forward for its gene therapy, MOGENRY. Japan's drug regulator is called the PMDA. It has agreed to review the company's application to market MOGENRY and has given it priority review. That means the review will move faster than usual.MOGENRY is being developed to treat a group of rare eye diseases. These are called inherited retinal dystrophies, or IRDs. This group includes retinitis pigmentosa (RP), the main cause of vision loss in Usher syndrome.
Most gene therapies are built to fix one specific gene mutation. That means they can only help patients whose vision loss comes from that gene. MOGENRY works differently. Instead of fixing the original genetic problem, it delivers a light-sensing protein into other retinal cells. These cells usually survive even after the original light-sensing cells die. This lets the surviving cells detect light on their own. As a result, vision could be restored regardless of which gene caused the damage. Nanoscope calls this a disease-agnostic therapy.
The application is backed by a clinical trial called RESTORE. The trial tested MOGENRY in patients with RP. Patients showed improved vision after one year. Vision was still improved about a year and a half later. The therapy also appeared safe, with no serious side effects reported. The U.S. FDA has also accepted a similar application for MOGENRY. That application would treat RP with severe vision loss, and it is still under review.
What this means for the Usher syndrome community: This is encouraging news for our community. If you or someone you love has Usher syndrome, you know it can be caused by many different genes. That is part of what makes it so hard to treat. MOGENRY takes a different path. Because it is designed to work no matter which gene is involved, it could one day help a much wider group of patients than a gene-specific treatment. MOGENRY is not yet approved, and work continues in both Japan and the United States. But this priority review is a step forward, one more sign that help is getting closer.
On Well-Being: The Whole Story
Victor Lombrana, USH2A "For a long time, it was easy to look at my life through the lens of what Usher syndrome has taken away. But I am learning that my story is much bigger than what I have lost. I am a husband, a father, a runner, a professional, and a person who still has goals, questions, dreams, frustrations, and hopes for the future. Usher syndrome has changed the way I experience the world, but it hasn’t taken away my desire to experience it. I still want to challenge myself, learn, accomplish things, be there for my family, and keep moving forward. Some days that looks different than it did when I was younger. That’s okay. Adaptation isn’t giving up—it’s finding another way forward. Reclaiming my story means allowing myself to acknowledge the difficult parts without allowing them to become the entire story. I don’t want people to see only a diagnosis when they see me. I want them to see Victor. A husband. A father. A runner. A professional. A person who is still growing, still learning, and still becoming. Usher syndrome is part of my story. It is not the whole story. Whether navigating changes in vision and hearing or advocating for loved ones, our strength lies in learning, adapting, and building resilience together. Usher syndrome is one thread in a much broader, richer story." To read the stories, visit the link here. 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.
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Meet Our 2027 Guide Dogs of USH!
The votes are in! After a community-wide vote, 12 hard-working guide dogs have been selected for our 2027 Guide Dogs of USH Calendar. Check out the photo collage below to meet this year's pups.
Thank you to everyone who sent in photos and voted. Every dog who entered appears on the calendar cover, because each one helped make this project special.
Key Dates:
November 17: The calendar goes on sale in the USH Shop.
December 9: Last day to order for holiday delivery.
A Gift That Gives Back
Each month features a guide dog alongside one way to connect with the Usher syndrome community, from joining the USH Trust to learning about current research. It makes a great holiday gift for dog lovers, family, and friends. All profits help the Usher Syndrome Coalition find and support every person with Usher syndrome around the world.
Mark your calendar for November 17!
Image Description: The cover of the 2027 Guide Dogs of USH Calendar, on a navy background. At the top, the title “Guide Dogs of USH” is in bold white letters. Below the title is a grid of photos of the 12 winning guide dogs. Under the grid, on the left, “2027” appears in large gold numbers above the web address usher-syndrome.org in white. Next to it are ten smaller photos of the other dogs who entered the contest, arranged like scattered snapshots with white borders, along with a yellow card that reads “Thanks to all our pups!” The Usher Syndrome Coalition logo is in the bottom right corner.
USH Tip
Different kinds of flashlights for winter months navigation with low vision:
Hands-Free & Wearable:
- Neck Lights: Flexible U-shaped lights that rest on the neck for hands-free illumination while reading or doing tasks.
- Headlamps: Tilting beam worn on the head to keep hands completely free for canes or guide dogs.
- Wrist / Pendant Lights: Wearable options that stay accessible and prevent dropping or misplacing the light.
Handheld & Task:
- COB LED Work Lights: Provide wide, even flood illumination rather than a harsh center hotspot.
- High-Lumen Tactical Lights: Offer bright, high-contrast zoom beams for navigating pitch-black outdoors or managing night blindness.
- Penlights: Slim, lightweight lights for targeted, short-range spot illumination (reading labels, keyholes).
Specialty & Multi-Use:
- Illuminated Magnifiers: Built-in LEDs paired with magnification to boost contrast and text size together.
- Spectacle / Cap Clips: Small lights that clip directly onto glasses frames or hat brims to point wherever you look.
- Foldable Desk Lamps: Rechargeable, portable lamps that fold flat for tabletop work anywhere.
Essential Features: High-contrast daylight spectrum LEDs (5000K–6000K), tactile/high-visibility buttons, adjustable brightness settings, and USB-C rechargeability.
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.
