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 ...
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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.
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A step closer to understanding Rett syndrome
Researchers at the Josep Carreras Leukaemia Research Institute revealed a new aspect of Rett Syndrome’s pathogenesis that can ...
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China Focus: China launches its first AI model for rare disease diagnosis
A visitor tries a surgery robot at the booth of Medtronic at the Medical Equipment & Healthcare Products Exhibition Area ...
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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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 ...
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 ...
Neuren secures FDA Fast Track designation for NNZ-2591 in Pitt Hopkins syndrome
Neuren Pharmaceuticals (ASX: NEU) has announced that the US Food and Drug Administration (FDA) has granted Fast Track ...