Rett Sendromu Tedavisinde Yeni Bir Umut: Trofinetid

Abstract

Trofinetid, Rett Sendromu'nun tedavisinde kullanılan bir ilaçtır. Mart 2023'te Amerika Birleşik Devletleri tarafından yetişkinlerde ve 2 yaş ve üstü çocuklarda Rett Sendromu tedavisi için onaylanmıştır. İlaç, insulin benzeri büyüme faktörü-1 (IGF-1)'in N-terminal tripeptid türevidir ve oral, küçük molekül, sentetik bir analogdur. FDA'nın trofinetidi onaylaması, Rett Sendromu için ilk yetkilendirilmiş tedaviyi işaret etmektedir.

Keywords

• Rett Sendromu
• Tedavi
• Trofinetid

A New Hope in the Treatment of Rett Syndrome: Trofinetide

Abstract

Trofinetide is a medication used to treat Rett Syndrome. It was approved by the United States in March 2023 for the treatment of Rett Syndrome in adults and children ages 2 and older. The drug is an N-terminal tripeptide derivative of insulin-like growth factor-1 (IGF-1) and is an oral, small molecule, synthetic analogue. The FDA's approval of trophinetide marks the first authorized treatment for Rett Syndrome.

Keywords

• Rett Syndrome
• Treatment
• Trofinetide

References

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