To date, Rett syndrome (RTT), a genetic disorder mainly caused by mutations in the X-linked
MECP2 gene, is increasingly considered a broad-spectrum pathology, instead of just a …

Introduction Rett syndrome is a rare genetic neurodevelopmental disorder that
predominantly impacts females. It presents with loss of acquired skills, impaired …

Trofinetide is the first drug approved by the FDA to treat Rett Syndrome in children aged 2
years or above. The drug significantly improved Rett syndrome behavioral scores Rett …

Rare diseases (RDs) represent a large and heterogeneous group of low-prevalence
conditions, and 473 million people could be affected worldwide. A total of 71.9% of these …

Peptide drugs generally show high performance in terms of safety, target affinity, and
efficacy, but have limitations such as low bioavailability and short half-life. To improve this …

El síndrome de Rett es un trastorno genético raro que afecta principalmente a las niñas,
causado por mutaciones en el gen MECP2. Generalmente se presenta en la primera …

With 54 new drugs and seven cellular and gene therapy products, the approvals by the US
Food and Drug Administration (FDA) recovered 2023 from the 2022 dent back to the levels …

Individuals with intellectual disability, including children with neurodevelopmental disorders,
are commonly affected by sleep disturbances. These sleep issues may manifest as typical …