Scientists at St. Jude Children's Research Hospital have found they can reverse the effects of HNRNPH2-related ...

Preclinical results from St. Jude Children’s Research Hospital show that an RNA-targeted therapy can restore function in a ...

NEW YORK, Jan. 08, 2024 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for ...

Tuesday, Taysha Gene Therapies Inc (NASDAQ:TSHA) shared initial clinical data from the first pediatric patient from the REVEAL Phase 1/2 adolescent and adult trial of TSHA-102, an investigational gene ...

Alterations in the GABAergic pathway in the brain contribute to the pathophysiology of neurodevelopmental disorders, including autism spectrum disorder and Angelman syndrome. Targeting the GABA-A ...

ST. LOUIS, April 17, 2026 /PRNewswire/ -- A decades-long dream, years in the making, Mercy Kids Center for Neurodevelopment in Chesterfield is celebrating its official opening, marking a new era of ...

Estimating that ReNU2 syndrome could account for around 10 percent of recessive neurodevelopmental disorder cases with a ...

Bringing clarity to families and paths to future gene therapies Histogram of log-scale U2-2 RNA levels in unaffected ...

If approved, UX111 will be the first approved therapy for the treatment of Sanfilippo syndrome Type A, a rare disease affecting young children that leads to progressive, irreversible neurodegeneration ...