David Lieberman, MD, PhD: The typical age [of diagnosis] is around 3 years old. Right now, we base it on a clinical diagnosis. That means we review the developmental history, and [determine] if they ...

Rett syndrome is one of the rare genetic disorders and almost exclusively affects girls. In Germany, an estimated 5,000 ...

Breakthrough Therapy designation granted based on FDA’s review of available clinical evidence of safety and efficacy from all 12 patients treated ...

Rett syndrome is a rare genetic disease affecting 1 in 10,000 newborn girls, characterised by sudden regression around 1 year of age, with loss of acquired language and motor skills and leading to ...

When evaluating the significance of some intervention, randomized-control trials are our gold standard. However, randomized-control trials are usually constrained by time and hindered by some bias. In ...

Shares of Taysha Gene Therapies, Inc. (TSHA) rallied 49% by Thursday morning after the U.S. Food and Drug Administration ...

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 ...

Acadia Pharmaceuticals announced last Friday that the FDA approved DAYBUE (trofinetide) for the treatment of Rett syndrome in adult and pediatric patients two years of age and older. DAYBUE is the ...

Rett syndrome is a rare neurological condition that disrupts physical and linguistic development, affecting around one in 10,000 women and even fewer men. For decades, researchers assumed that people ...

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 ...