Please provide your email address to receive an email when new articles are posted on . “To our knowledge, this is the first targeted therapy for achondroplasia that has resulted in a highly ...

Credit: BioMarin. The expanded approval was based on data from a phase 2 trial that included children less than 5 years of age with achondroplasia. The Food and Drug Administration (FDA) has expanded ...

The oral FGFR1-3 selective tyrosine kinase inhibitor infigratinib was found to be safe and effective at the highest studied dose among children with achondroplasia in a phase II dose-finding study.

M M Faruqui, known professionally as Lilliput, is a renowned actor who has appeared in numerous plays, movies, and TV series. He was recently seen playing Dadda Tyagi in Mirzapur Season 2 and gained ...

The fitness world is always evolving, and today we’re focusing on a crucial topic: how people with achondroplasia can enjoy an active and healthy lifestyle. Achondroplasia is a genetic condition ...

Genetics can seem dry when the real world applications are not made apparent. Genetics lies at the essence of life as we know it, and when there is a kink in the system, it can severely affect the ...

The International Day of Persons with Disabilities (IDPD) is observed every year on December 3rd to promote awareness of disability issues. It was established by the United Nations General Assembly in ...

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A Prescription Drug User Fee Act target date of November 30, 2025 has been assigned to the application. The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application ...

The results are preliminary, but a novel investigative agent has shown promise in a small trial in children for the treatment of achondroplasia, the most common form of human dwarfism. The side effect ...