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
References
- 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.
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
References
- 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.
Details
| Primary Language | English |
|---|---|
| Subjects | Psychiatry |
| Journal Section | Letter To The Editor |
| Authors | |
| Early Pub Date | October 22, 2024 |
| Publication Date | November 1, 2024 |
| Submission Date | April 4, 2024 |
| Acceptance Date | June 24, 2024 |
| Published in Issue | Year 2024 Volume: 6 Issue: 3 |
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