Advancing therapeutic approaches for Rett syndrome: investigating trofenitide's role-a comprehensive review

Nikita Gupta; Kachakayala Ramya; Kommuri C. Rao; Syeda M. Fatima; Khadija A. Siddiqui

doi:10.18203/2394-6040.ijcmph20242191

Authors

Nikita Gupta Department of Pharmacy Practice, St Paul’s College of Pharmacy, Hyderabad, Telangana, India
Kachakayala Ramya Department of Pharmacy, St. Paul’s College of Pharmacy, Hyderabad, Telangana, India
Kommuri C. Rao Department of Pharmacy, St. Paul’s College of Pharmacy, Hyderabad, Telangana, India
Syeda M. Fatima Department of Pharmacy, St. Paul’s College of Pharmacy, Hyderabad, Telangana, India
Khadija A. Siddiqui Department of Pharmacy, St. Paul’s College of Pharmacy, Hyderabad, Telangana, India

DOI:

https://doi.org/10.18203/2394-6040.ijcmph20242191

Keywords:

Rett syndrome, Methyl-Cpg-binding protein 2, Histone deacetylase, Nuclear corepressor, Trofenitide

Abstract

Rett syndrome is a complex neurodevelopmental disorder that mainly affects girls. It involves the loss of hand skills, walking difficulties, breathing problems, seizures, and repetitive hand movements. The syndrome starts with normal development but then goes through a regression, causing significant disability. It is caused by genetic mutations in the methyl-Cpg-binding protein 2 (MECP2) gene on the X chromosome. Rett syndrome poses challenges for patients and their families, requiring comprehensive support and a multidisciplinary approach involving healthcare professionals, educators, and social support networks. Ongoing research on trofenitide in Rett syndrome focuses on understanding its optimal dosing and long-term efficacy. Recent studies have revealed its neuroprotective and neurotrophic properties, suggesting involvement in important signaling pathways for neuronal development and synaptic plasticity. Future research aims to uncover specific molecular targets and pathways influenced by trofenitide, enabling the development of more personalized treatment strategies. Additionally, exploring the synergistic effects of trofenitide with other therapies, such as histone deacetylase (HDAC) inhibitors and N-methyl D-aspartate (NMDA) receptor modulators, shows promise for improved outcomes. Precision medicine approaches utilizing molecular profiling may aid in patient stratification and the development of targeted therapies.

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