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.

Romi Loffler fears she will be among the hardest hit by East Renfrewshire Council's decision to means-test and charge for ...

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 ...

If you haven't heard of Rett syndrome, it's probably because it's very rare. Affecting an estimated one in 12,000 baby girls ...

The field of neuroscience has benefited significantly from the groundbreaking work of dedicated researchers who have ...

Metabolic syndrome is a constellation of conditions that together increase the risk of type 2 diabetes, stroke, and cardiovascular problems, including heart attack. The features of metabolic syndrome ...

She was 21 when she died from complications of Rett syndrome on Dec. 14, 2024. “She’s the bravest person I know,” Endres said. For him and his brother Griffin, there are good days and there ...

including Rett syndrome, and other central nervous system (CNS) disorders, announced today it was issued a new U.S. Patent No. 12,180,174 entitled “A2-73 CRYSTALLINE POLYMORPH COMPOSITIONS OF ...

Taysha Gene Therapies' TSHA-102 tackles Rett syndrome’s root cause with its miniMECP2 transgene and scAAV delivery. So far, this is TSHA’s main value driver, and it appears safe and effective ...