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…
The Importance of Braille For any child with a rare disease that leads to blindness or visual impairment such as Stargardt disease, Usher Syndrome, Alström syndrome, or Retinal Blindness life…
Stargardt Disease Stargardt disease is a rare condition that causes vision loss due to the buildup of vitamin A byproducts in the retina. The byproduct causing the largest buildup is…
May is Healthy Vision Month! Rare disease or not, maintaining healthy eye health should be a priority for all of us. The National Institute of Health has resources for not…
Stargardt Disease Stargardt disease is a rare disease which results in progressive vision loss. It is also referred to as juvenile macular degeneration. Most often, the disease presents during childhood.…
The longer patients live without a diagnosis, the longer they are living without the proper care and the proper treatment. The problem is twofold when a patient is diagnosed incorrectly.…
The United States Food and Drug Administration has awarded twelve research grants worth a total of over $18 million for research into rare diseases. For more information about this news,…
According to a story from Eye on the Cure, the biopharmaceutical company Ophthotech is hard at work researching and developing gene therapy solutions for a variety of rare retinal conditions.…
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