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More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings highlight the need for...
Quinnipiac University held a symposium on March 24 at the North Haven campus to help bring awareness to rare diseases. The ninth annual Rare Disease Day Symposium was presented by the Frank H. Netter MD School of Medicine at Quinnipiac. Attendees were able to join in-person or virtually. The symposium...
ASHBURN, Va. — Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX), a specialty pharmaceutical company exclusively focused on the development and commercialization of innovative therapies that treat rare and orphan diseases, today announced it has filed U.S. and International patent applications for a novel combination product as a potential treatment of Netherton Syndrome....
NANTONG and SUZHOU, China — Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to RAG-18, one of the company’s lead saRNA product candidates. This designation follows the recent Rare Pediatric Disease...
Keira Mathewson is a very cute little girl, two months shy of her second birthday. She also has a brain disease that slows her development, reduces her movement and affects her speech. Keira, who lives with her parents Julie Lewis and Michael Mathewson at Brassall, will be one of the...
Reston, VA — Radioguided surgery can detect and remove metastatic pelvic lymph nodes in patients newly diagnosed with prostate cancer, according to research published in the March issue of The Journal of Nuclear Medicine. Targeting the prostate-specific membrane antigen (PSMA), which is overexpressed in most prostate cancer patients, radioguided surgery can improve...