Latest news with #StargardtDisease
BBC News
19-05-2025
- Entertainment
- BBC News
Blind woman to hold fashion show on Yr Wyddfa summit
"I can see that you are there, but with the light behind you, I can't see your face, it's just a blur."Tilly Dowler is describing what she can see when she looks at was registered blind in 2022 and has completely lost her central vision because of a genetic condition called Stargardt this, she has decided to climb Yr Wyddfa, also known as Snowdon, the highest mountain in Wales, with five other blind or partially sighted walkers to raise money for they reach the summit, the 26-year-old is hosting a sustainable fashion show. "We wanted to do something crazy and make people think, wow, why are they doing that?" said Tilly, who is from Castle Donington in challenge, named Sightless on Snowdon, aims to raise funds for Leicestershire-based charity Vista, which Tilly credits with supporting her through sight loss."When you're diagnosed with sight loss, it's like it's the end of the world and you panic and you think that no one is going to be there to understand or help you."At Vista, everyone understands and they are all really helpful, so I wanted to give back."They didn't only help me, they helped my mum. They got me in contact with other organisations that would help, and they are very close to my heart," she who started to lose her sight aged 15, said the charity had given her the confidence and training to use a cane. 'Trailblazer' Tilly's plan to put on a show at the top of the mountain in Wales comes from her love of pre-loved clothes shop Wanted Wardrobe in Castle Donington donates 10% of its profits to sight loss charities."We are very aware that the weather might not be great so what we're going to do is take lots of different clothes in lots of different sizes, head pieces, accessories."We're going to pop them on even if it means over our jackets and just give a little bit of a fashion show."The group of walkers are taking on the mountain on 10 June and have sighted guides to help them along the to Vista, one in five people are expected to lose their sight across the UK every year, affecting 35,500 people across Leicester, Leicestershire and Wright, CEO of Vista, said: "Tilly is a trailblazer for others living with sight loss. "We are so grateful for her ongoing support and will be supporting her all the way to the summit of Snowdon and beyond."
Yahoo
29-04-2025
- Business
- Yahoo
Belite Bio Announces Poster Presentations at the Association for Research in Vision and Ophthalmology 2025 Annual Meeting
SAN DIEGO, April 29, 2025 (GLOBE NEWSWIRE) -- Belite Bio, Inc. (NASDAQ: BLTE), a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announced that the Company will give two poster presentations at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting being held May 4–8, 2025, in Salt Lake City, Utah. The presentations will highlight baseline patient characteristics from the ongoing DRAGON trial and pharmacokinetic and pharmacodynamic properties from the ongoing DRAGON II trial, both in Stargardt disease. Poster Presentations: Title: Baseline Characteristics of Adolescent Stargardt Disease Subjects Participating in a Phase 3 Study of Tinlarebant (DRAGON Trial)Presenting Author: Dr. Ruifang SuiPresentation Number: 1463Session Number: 215Session Title: Retinitis Pigmentosa and IRD ISession Date/Time: Monday, May 5, 8:30-10:15 a.m. MDT Title: Pharmacokinetic and Pharmacodynamic Properties of an Oral Investigational Treatment for Stargardt Disease in Adolescent Japanese PatientsPresenting Author: Dr. Kaoru FujinamiPresentation Number: 1431Session Number: 215Session Title: Retinitis Pigmentosa and IRD ISession Date/Time: Monday, May 5, 8:30-10:15 a.m. MDT About Tinlarebant (a/k/a LBS-008)Tinlarebant is a novel oral therapy that is intended to reduce the accumulation of vitamin A-based toxins (known as bisretinoids) that cause retinal disease in Stargardt Disease type 1 (STGD1) and also contribute to disease progression in geographic atrophy, or advanced dry age-related macular degeneration (AMD). Bisretinoids are by-products of the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye. Tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), the sole carrier protein for retinol transport from the liver to the eye. By modulating the amount of retinol entering the eye, Tinlarebant reduces the formation of bisretinoids. Tinlarebant has been granted Fast Track Designation and Rare Pediatric Disease designation in the U.S., Orphan Drug Designation in the U.S., Europe, and Japan, and Sakigake Designation in Japan for the treatment of STGD1. Stargardt Disease (STGD1)STGD1 is the most common inherited macular dystrophy (causing blurring or loss of central vision) in both adults and children. The disease is caused by mutations in a retina-specific gene (ABCA4), which results in progressive accumulation of bisretinoids leading to retinal cell death and progressive loss of central vision. The fluorescent properties of bisretinoids and the development of retinal imaging systems have helped ophthalmologists identify and monitor disease progression. Currently, there are no FDA approved treatments for STGD1. Importantly, the appearance of bisretinoids, followed by retinal cell death, and progressive loss of vision is also observed in geographic atrophy (GA) patients. Therefore, Belite Bio is evaluating safety and efficacy of Tinlarebant in GA patients in a 2-year Phase 3 study (PHOENIX). About Belite BioBelite Bio is a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, such as Stargardt disease type 1 (STGD1) and Geographic Atrophy (GA) in advanced dry age-related macular degeneration (AMD), in addition to specific metabolic diseases. Belite's lead candidate, Tinlarebant, an oral therapy intended to reduce the accumulation of toxins in the eye, is currently being evaluated in a Phase 3 study (DRAGON) and a Phase 2/3 study (DRAGON II) in adolescent STGD1 subjects and a Phase 3 study (PHOENIX) in subjects with GA. For more information, follow us on Twitter, Instagram, LinkedIn, Facebook or visit us at Media and Investor Relations Contact:Jennifer Wu ir@ Julie Fallonbelite@ in to access your portfolio
