Rett syndrome is a rare developmental disorder, most often due to a mutation in the MECP2 gene on the X chromosome, but it’s not usually inherited. Advances in genetics have launched a new era in ...
The 2022 Option Agreement between Astellas and Taysha has expiredRegaining full rights to TSHA-102 Rett syndrome program enables Taysha to focus ...
Taysha Gene Therapies shares fall after regaining full rights to Rett syndrome drug TSHA-102 and finalizing FDA alignment for ...
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 ...
SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals, Inc. (Nasdaq: ACAD) today announced that interim data from the open-label real-world LOTUS™ study will be presented at the 2024 International Rett ...
In a comprehensive preclinical program, NGN-401 demonstrated robust therapeutic and safety benefits, delivering MECP2 to key brain regions affected by Rett syndrome NGN-401 utilizes Neurogene’s ...
NEW YORK and TRUMBULL, Conn., April 30, 2025 /PRNewswire/ -- Apertura Gene Therapy, a biotechnology company focused on innovative gene therapy solutions, and the Rett Syndrome Research Trust (RSRT), ...
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 ...
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 ...
Results that may be inaccessible to you are currently showing.
Hide inaccessible results
Feedback