FDA alignment achieved on pivotal study design supporting a planned Biologics License Application (BLA) submission -- 30-patient pivotal study initiated, evaluating seizure reduction and ...

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 ...

The superimposed green lines denote five affected individuals with recessive ReNU2 syndrome. The stark difference between affected and unaffected individuals enables accurate diagnostics and points to ...

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the ...

Three studies published in Nature Genetics look at the identification of a common recessive neurodevelopmental disorder (NDD).

The vehicles that deliver gene editing cargo to the body’s cells can be toxic, complicating gene therapies, particularly those addressed to brain cells of developing fetuses. Less toxic carriers, ...

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 ...

New research urges reclassifying schizophrenia as a neurodevelopmental syndrome—advancing diagnosis, care, and public ...

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 ...

Histogram of log-scale U2-2 RNA levels in unaffected individuals (grey). The superimposed green lines denote five affected individuals with recessive ReNU2 syndrome. The stark difference between ...