A New Hope in the Treatment of Rett Syndrome: Trofinetide

Yıl 2024, Cilt: 6 Sayı: 3, 134 - 135, 01.11.2024

Doğancan Sönmez

https://doi.org/10.38175/phnx.1465288

Öz

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.

Anahtar Kelimeler

Rett Syndrome, Treatment, Trofinetide

Rett Sendromu Tedavisinde Yeni Bir Umut: Trofinetid

Öz

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.

Anahtar Kelimeler

Rett Sendromu, Tedavi, Trofinetid

Kaynakça

• Petriti U, Dudman DC, Scosyrev E, Lopez-Leon S. Global prevalence of Rett syndrome: systematic review and meta-analysis. Systematic reviews. 2023;12:5.
• Neul JL, Percy AK, Benke TA, Berry-Kravis EM, Glaze DG, Peters SU, et al. Trofinetide Treatment Demonstrates a Benefit Over Placebo for the Ability to Communicate in Rett Syndrome. Pediatric neurology. 2024;152:63-72.
• Neul JL, Percy AK, Benke TA, Berry-Kravis EM, Glaze DG, Marsh ED, et al. Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study. Nat Med. 2023;29:1468-75.
• Abbas A, Fayoud AM, El Din Moawad MH, Hamad AA, Hamouda H, Fouad EA. Safety and efficacy of trofinetide in Rett syndrome: a systematic review and meta-analysis of randomized controlled trials. BMC pediatrics. 2024;24:206.
• Furqan M. Trofinetide-a new chapter in rett syndrome’s treatment. Frontiers in pharmacology. 2023;14:1284035.