An expert explains how Rett syndrome is diagnosed, how commonly it is misdiagnosed, disease progression, and which providers are best positioned to manage a Rett Syndrome patient. David Lieberman, MD, ...
In Rett syndrome, a genetic condition affecting girls almost exclusively, mutations in the MECP2 gene cause a regression of language and motor skills starting at 12 to 18 months of age. Children then ...
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 ...
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 ...
David Lieberman, MD, PhD, describes Rett syndrome, including the various stages, incidence, and potential causes of this disease. David Lieberman, MD, PhD: Rett syndrome is a neurodevelopmental ...
NEW YORK--(BUSINESS WIRE)--Neurogene Inc., a company founded on the vision to push the boundaries of genetic medicine to address complex and devastating neurological diseases, announced the addition ...
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 ...
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 ...
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