"Researchers at the National Institutes of Health (NIH) have developed eye drops that extend vision in animal models of a group of inherited diseases that lead to progressive vision loss in humans, known as retinitis pigmentosa. The eye drops contain a small fragment derived from a protein made by the body and found in the eye, known as pigment epithelium-derived factor (PEDF).?PEDF helps preserve cells in the eye’s retina. A report on the study is published in?Communications Medicine."
USHER 1F COLLABORATIVE INC
研究服务
NEWTONVILLE，Massachusetts 135 位关注者
Our mission is to fund medical research to find an effective treatment to save the vision of those with Usher 1F.
关于我们
USHER 1F COLLABORATIVE INC is a company based out of 321 WALNUT ST NO 228, NEWTONVILLE, Massachusetts, United States.
- 网站
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https://usher1f.org
USHER 1F COLLABORATIVE INC的外部链接
- 所属行业
- 研究服务
- 规模
- 2-10 人
- 总部
- NEWTONVILLE，Massachusetts
- 类型
- 非营利机构
- 创立
- 2013
地点
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主要
获取路线
321 WALNUT ST NO 228
US，Massachusetts，NEWTONVILLE，02460
USHER 1F COLLABORATIVE INC员工
动态
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From Usher 1F Collaborative board member Frank Gentile, PhD: Regeneron yesterday reported good results with their gene therapy program for children with mutations in otoferlin. This was a program they got from their acquisition of Decibel in 2023. 10/11 children (some as young as 10 months old) had hearing improvement. I was lucky enough to work on the build and launch of Decibel in 2015 when I was at Third Rock. This is life altering for these children and validation to what we are trying to do with Usher 1F.
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Calling all rare disease community members – patients, caregivers, clinicians, researchers, and advocates to join a petition to Congress in honor of Rare Disease Week on Capitol Hill 2025. ?On behalf of our nation’s rare disease community, the petition urges Congress to continue their support of steady and robust federal agency leadership, federal biomedical research funding, and public health agency resources. You can join the efforts by filling out the form to the right to sign the following petition. Add your name today. Then encourage your family and friends to join. The Community’s Letter will be sent to all Members of Congress on Rare Disease Day, Friday, February 28th. Your first and last name will appear on the petition sent to Congress. Individual names will not be shared publicly.
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Our 2024 Annual Report published today - take a look at our great accomplishments last year! https://lnkd.in/erhzA6Ry
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We're proud and excited to be featured in the Chan Zuckerberg Initiative Rare As One Network Cycle 1 Impact Report! https://lnkd.in/ePB28i6t See page 70 for the write-up on our accomplishments during and since the grant period.
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Today is #GivingTuesday. Our scientists are hard at work in labs funded by Usher 1F Collaborative, seeking a cure for everyone who faces a future of blindness due to Usher 1F. Please join us. https://lnkd.in/efYbyE-V