Taysha Gene Therapies Inc. (NASDAQ:TSHA) is one of the best penny stocks to buy for 2026. On January 6, the company confirmed ...

In the comfort of her Miami home, 34-year-old Julianne Espinosa lies in bed captivated by videos of Disney princesses flashing across her television screen. As echoes of “The Little Mermaid” fill her ...

The neurodevelomental disease Rett syndrome is usually caused by mutations in a gene called MECP2, which is located on the X chromosome. Patients lose coordination, mobility, and their ability to ...

First patient dosed in REVEAL pivotal trial evaluating TSHA-102 (N=15, aged 6 to <22 years) in Q4 2025, with enrollment advancing across ...

Rett syndrome is a neurodevelopmental disorder that primarily affects girls, most of whom have mutations in the transcription regulatory gene MECP2. However, mutations in MECP2 also have been ...

UAB will study a drug originally developed for Parkinson’s disease that may help reduce breath holding in patients with Rett syndrome. Pediatric neurologist Alan Percy, M.D., is a leading clinician ...

Credit: Acadia. Daybue Stix, a strawberry-flavored trofinetide powder mixable with water-based liquids, gains FDA approval for Rett syndrome treatment. Acadia expects to have the new formulation ...

Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today provided an update on ...

SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the company has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) ...

SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that Health Canada has granted marketing authorization of DAYBUE™ (trofinetide) for the treatment of Rett ...