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

A New Hope in the Treatment of Rett Syndrome: Trofinetide

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

Kaynakça

1. Petriti U, Dudman DC, Scosyrev E, Lopez-Leon S. Global prevalence of Rett syndrome: systematic review and meta-analysis. Systematic reviews. 2023;12:5.
2. 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.
3. 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.
4. 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.
5. Furqan M. Trofinetide-a new chapter in rett syndrome’s treatment. Frontiers in pharmacology. 2023;14:1284035.