Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
At the very top of the Rensselaer Polytechnic Institute's website reads a bold statement and call to action: "Why not change the world?" As one of the United States' most…
ABCA4-associated retinopathies are a group of conditions that cause progressive macular degeneration and vision loss due to ABCA4 mutations. Stargardt disease, cone-rod dystrophy type 3 (CORD3), and retinitis pigmentosa 19…
According to an article from Yahoo! Finance, biopharmaceutical company Belite Bio, Inc. has initiated the Phase 3 DRAGON clinical study to evaluate LBS-008 for individuals with Stargardt disease. The…
An article published by Ophthalmology Times Europe shared that the first patient had been dosed in the Phase 2 STARLIGHT clinical trial evaluating MCO-010 as a potential therapeutic option…
Inherited retinal diseases (IRDs) are a group of genetic ocular disorders characterized by vision loss or blindness. A few examples are retinitis pigments (RP) and Usher syndrome. There has been…
Ala Moshiri from the University of California is both a researcher and an eye doctor. He recently discussed his frustration surrounding the lack of available treatments for so many genetic…
The Food and Drug Administration (FDA) has four distinct approaches to expedite the availability of therapies for rare, severe, or life-threatening illnesses: Priority Review, Breakthrough Therapy, Fast Track, and Accelerated…
According to a recent article, a mother was able to see her baby daughter clearly after being diagnosed with the rare Stargardt disease. Stargardt Disease Stargardt disease is an inherited…
Sometimes, companies work together to develop the best therapeutic options for patients. According to Optical Prism, biopharmaceutical company reVision Therapeutics, Inc. and specialty pharmaceutical company Aequus Pharmaceuticals Inc. recently joined…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
On July 14, 2021, biopharmaceutical company Alkeus Pharmaceuticals, Inc. ("Alkeus") shared via press release that its therapy ALK-001 (C20-D3-vitamin A) received Breakthrough Therapy designation from the FDA for the treatment…
George Mendoza has inspired others throughout his whole life. He has been an athlete, an author, a motivational speaker, and more. Born in 1955 in New York City, Mendoza has…
As reported in AP News, Bradford and Bryan Manning broke into a market in the fashion industry often overlooked: the blind. The glitz of fast fashion doesn’t work. So what are…
As reported in Medical Xpress; Fight for Sight, a charity in the UK dedicated to vision, has an exciting announcement for people with macular diseases: they have just granted funding…
Bryan and Bradford Manning recently gave an interview to the Associated Press. The brothers told the story of their vision loss as a result of a rare degenerative eye disease.…
According to a story from PR Newswire, the biopharmaceutical company reVision Therapeutics, Inc. has just announced that its experimental therapy REV-0100 has received both Rare Pediatric Disease designation and Orphan…
According to a story from BioSpace, the US Food and Drug Administration (FDA) recently announced that it has awarded grant funding that will go towards six clinical trials that will…
The United States FDA has just announced that they will be providing 6 new grants, which are worth 16 million dollars for rare disease research. These grants will last a…
Cornell University and reVision Therapeutics have come together through a licensing agreement to develop a treatment for Stargardt disease, according to BioSpace. They will be working on and commercializing Cornell's…
Vision loss is a major symptom of Stargardt disease, so a main goal of treatments is to combat this loss. In order to test the effectiveness of these treatments, there…
Macular disease or stargardt disease, is a rare condition whose onset can occur at anytime. It results in damage to the retina, though most patients do not become completely blind.…
As reported in Globe News Wire, amidst a difficult time for rare disease patients as the medical world’s refocuses on the coronavirus, a new scholarship has been announced that could…
Kubota Vision, a subsidiary of Kubota Pharmaceutical Holdings, has recently finished enrollment in the third phase of their emixustat hydrochloride (emixustat) trial. Emixustat hydrochloride is a treatment for Stargardt disease…
According to a story from Financial Buzz, the ophthalmology company Acucela Inc. has recently announced an update on the progress of its phase 3 clinical trial. This clinical trial is…
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