Based on China's accumulated rare disease knowledge and genetic data from its population, PUMCH-GENESIS is the world's first ...

Based on China's accumulated rare disease knowledge and genetic data from its population, PUMCH-GENESIS is the world's first ...

Rett syndrome is a rare genetic disease affecting 1 in 10,000 newborn girls, characterized by sudden regression around 1 year ...

Building on more than two decades of research, a study by MIT neuroscientists at The Picower Institute for Learning and Memory reports a new way to treat pathology and symptoms of fragile X syndrome, ...

Discover Stoke Therapeutics' innovative RNA-based treatments like zorevunersen for Dravet syndrome, with a promising pipeline ...

Phil and Claire Drinkwater have raised more than £11,000 for charity since the loss of their 'joyful' son Jude ...

Rare diseases have become an attractive target for pharmaceutical companies both on the ASX and internationally.

Anavex Life Sciences Corp. (NASDAQ: AVXL), a clinical-stage biopharmaceutical company focused on developing innovative treatments for Alzheimer's disease, Parkinson's disease, schizophrenia, ...

Company to host a webcast today at 8:30 a.m. Eastern Time NEW YORK, Feb. 12, 2025 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. ("Anavex" or the "Company") (Nasdaq: AVXL), a clinical-stage biopharmac ...

Company to host a webcasttoday at 8:30 a.m. Eastern TimeNEW YORK, Feb. 12, 2025 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the ...

Jones Trading initiated coverage on ProQR Therapeutics N.V. (NASDAQ:PRQR) with a Buy rating and set a price target of $11.00, representing significant upside from the current price of $2.19. The ...