Galila Yohannes’ parents, who originally hail from Eritrea, brought their family to Israel for a better life. And while they never would have expected what came next, this move likely…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
As the world becomes increasingly technological, researchers and scientists are leveraging these advances for scientific improvement. Artificial intelligence (AI) is now being used to develop products, screen drug candidates,…
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…
For many rare diseases, gene therapy, not traditional therapeutics, is perhaps the only means to significant relief and cure. However, the path to gene therapy has been strewn with obstacles…
In the United States, Fast Track designation is granted to drugs designed to treat rare and/or serious conditions and that fill an unmet medical need. The process aims to…
Three of Edith Lemay and Sebastien Pelletier’s four children were diagnosed with retinitis pigmentosa, an untreatable genetic condition. The parents were told their children would eventually be blind. In trying…
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…
In April 2022, Dr. Adrián Alambiaga successfully defended his doctoral thesis at CEU Cardenal Herrera University. His thesis centered around the exploration of progesterone as a way to stop cell…
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…
When Krista Webb was just two years old, doctors determined that she had some level of hearing loss. Her family was surprised about this; nobody else in her family, and…
ProQR Therapeutics is working to develop unique therapeutic solutions for genetic eye diseases. Currently, the company is evaluating its therapeutic candidate ultevursen within the Phase 2/3 Sirius and Celeste clinical trials; you can read…
If you would like to read the first part of this interview, please take a look at Part 1 here. In the first part of the interview, I spoke with…
Clinical trials, drug development, and medical research have the potential to change the therapeutic landscape for patients with a wide variety of conditions. At ProQR, founder and CEO Daniel de…
According to a news release from RNA therapeutics company ProQR Therapeutics N.V. ("ProQR"), the first patients have been dosed in two Phase 2/3 clinical trials evaluating QR-421a for USH2a-mediated retinitis…
Fast Track designation is requested by the drug company; this process and designation are designed to facilitate the development and review of therapies which can treat serious or underserved conditions,…
While some therapies offer potential benefits to patients, not all therapies show efficacy within the clinical trial environment. Recently, shares MedCityNews, this occurred with biotechnology company Biogen Inc.'s ("Biogen") gene…
Retinitis Pigmentosa Retinitis Pigmentosa is a rare condition which progressively causes vision loss. Genetic lesions which first affect the rod photoreceptors first cause vision loss at night. Next, cone photoreceptors are…
A variety of gene mutations cause retinitis pigmentosa (RP) - but how do those genes affect the degeneration of rod and cone photoreceptors in the eyes? According to Medical XPress,…
In a press release from early March 2021, cell therapy company CiRC Biosciences, Inc. ("CiRC") shared that its chemically induced photoreceptor-like cells (CiPCs) received Orphan Drug designation from the FDA.…
Recently, biopharmaceutical company GenSight Biologics ("GenSight") announced the publication of data from a study evaluating GS030-Drug Products (GS030-DP or "GS030") as a potential treatment option for retinitis pigmentosa (RP). Although…
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…
Researchers from Nanoscope, thanks to funding by the NIH, have developed a protein that can effectively restore vision in mice. This study was published in Nature Gene Therapy. The Study…
For patients who lose their vision, artificial and implantable devices like retinal prostheses offer a way forward. But while these prostheses are already somewhat effective, a Korean research team recently…
Janssen Pharmaceuticals has an ongoing Phase 1/2 clinical trial for the inherited retinal disease X-linked retinitis pigmentosa (XLRP). They are investigating an adeno-associated gene therapy called RPGR as a therapeutic…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
