Over the next 2-2.5 years, biotech spin-out company Augustine Therapeutics will be working to understand both the underlying biology of Charcot-Marie-Tooth disease (CMT) and potential therapeutic options for patients.…
Rare Disease Day each year on February 28th provides a time for the rare disease community to reflect on the progress of the year, the continued gaps in rare disease…
MSN News recently interviewed Dr. Laxmikant Palo, with the conversation focusing on the situations of people living with rare diseases in India and how to improve them. According to Dr.…
For over 15 years, researchers and scientists have been working to understand the underlying genetics of macular telangiectasia (MacTel), a rare and debilitating eye disease, through the MacTel Project.…
For years, scientists have known the mechanisms underlying normal, as well as the disease-causing (pathogenic) versions, of prions. But according to a recent article published in Sci-Tech Daily, no one…
As reported in PR NewsWire; this March, Calliditas Therapeutics AB announced they have submitted a New Drug Application (NDA) for their novel oral solution, Nefecon, to treat primary IgA Nephropathy…
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…
According to a study from hindawi.com, the serum glycocholic acid (GCA) to total bile acid (TBA) ratio can be used to identify people living with nonalcoholic fatty liver disease (NAFLD).…
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…
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