Autophagy malfunction study moves a step closer to understanding Rett syndrome
Rett syndrome is a rare genetic disease affecting 1 in 10,000 newborn girls, characterized by sudden regression around 1 year ...
Stoke Therapeutics: A Pivotal Moment For This RNA Pioneer
Discover Stoke Therapeutics' innovative RNA-based treatments like zorevunersen for Dravet syndrome, with a promising pipeline ...
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Taysha Gene Therapies Is Showing Promise For Rett Syndrome
In particular, its leading candidate is hands down TSHA-102 for Rett syndrome. The way this therapy works is by using “self-complementary” adeno viruses [scAAV] to deliver its treatment.
Taysha Gene Therapies to Release Full-Year 2024 Financial Results and Host Conference Call and Webcast on February 26
Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage biotechnology company focused on advancing adeno-associated ...
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Acadia Pharmaceuticals Submits Marketing Authorization Application to the European Medicines Agency for Trofinetide for the Treatment of Rett Syndrome
“The Rett syndrome community in Europe is excited by the prospect of having an available treatment option. Rett syndrome has a significant impact on people living with this condition, and also ...
Medscape10d
Rett Syndrome: Model of Neurodevelopmental Disorders
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 ...