Trofinetide for Rett Syndrome: Highlights on the Development and Related Inventions of the First USFDA-Approved Treatment for Rare Pediatric Unmet Medical Need

Rett syndrome (RTT) is a rare disability causing female-oriented pediatric neurodevelopmental unmet medical need. RTT was recognized in 1966. However, over the past 56 years, the United States Food and Drug Administration (USFDA) has authorized no effective treatment for RTT. Recently, Trofinetide w...

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Detalles Bibliográficos
Autores principales: Hudu, Shuaibu A., Elmigdadi, Fayig, Qtaitat, Aiman Al, Almehmadi, Mazen, Alsaiari, Ahad Amer, Allahyani, Mamdouh, Aljuaid, Abdulelah, Salih, Magdi, Alghamdi, Adel, Alrofaidi, Mohammad A., Abida, Imran, Mohd
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10420089/
https://www.ncbi.nlm.nih.gov/pubmed/37568516
http://dx.doi.org/10.3390/jcm12155114

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10420089/
https://www.ncbi.nlm.nih.gov/pubmed/37568516
http://dx.doi.org/10.3390/jcm12155114

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