A new gene-targeting strategy that boosts a crucial brain protein could pave the way for the first effective treatment for Rett syndrome. Researchers at Texas Children’s Duncan Neurological Research I ...
A team of researchers at Baylor College of Medicine and the Duncan Neurological Research Institute (Duncan NRI) at Texas Children's Hospital reports in Science Translational Medicine a potential new ...
A Phase I/IIa clinical trials co-led by Linda Laux, MD, from Ann & Robert H. Lurie Children's Hospital of Chicago, show that ...
The drug was finally approved in September 2025 through the FDA’s accelerated approval pathway, based on improvement in knee extensor muscle strength, for adults and children with Barth syndrome who ...
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