The novel first-line therapy for rett syndrome

Wania Sultan; Hamza Irfan; Kainat Shariq

doi:10.14419/zpn3s949

Article Details

The novel first-line therapy for rett syndrome

Authors:

Wania Sultan | Hamza Irfan | Kainat Shariq

Publisher:

Science Publishing Corporation

Journal:

International Journal of Medicine Vol.11 Issue 1, pp.15-16

Document Type:

Article

Publication Date:

12/2023

Document Language:

English

Document ID (Org No.):

2001388090

pISSN:

2309-1622

DOI:

10.14419/zpn3s949

Keywords:

]

Abstract:

 Rett Syndrome (RTT) is a neurodevelopmental disorder predominantly affecting females and characterized by hyperkinetic movement disorders and related symptoms. At present, the available treatments for RTT primarily focus on managing symptoms and providing supportive care. This article examines the emergence of trofinetide as an innovative therapeutic approach targeting the underlying molecular and synaptic abnormalities associated with RTT. The approval of trofinetide, marketed as DAYBUE™, as the primary treatment for Rett Syndrome by the US Food and Drug Administration (FDA) is based on encouraging outcomes from clinical trials. Ongoing open-label studies are currently underway to evaluate the long-term efficacy and safety of trofinetide in various age groups, thus providing valuable insights into its sustained benefits. These advancements in research, along with other potential treatments under investigation, offer promising prospects for the development of improved and personalized management strategies for individuals with Rett Syndrome.

Article Details

The novel first-line therapy for rett syndrome

Authors: Wania Sultan | Hamza Irfan | Kainat Shariq

Publisher: Science Publishing Corporation

Journal: International Journal of Medicine Vol.11 Issue 1, pp.15-16

Document Type: Article

Publication Date: 12/2023

Document Language: English

Document ID (Org No.): 2001388090

pISSN: 2309-1622

DOI: 10.14419/zpn3s949

]

Issues

Authors:

Wania Sultan | Hamza Irfan | Kainat Shariq

Publisher:

Science Publishing Corporation

Journal:

International Journal of Medicine Vol.11 Issue 1, pp.15-16

Document Type:

Article

Publication Date:

12/2023

Document Language:

English

Document ID (Org No.):

2001388090

pISSN:

2309-1622

DOI:

10.14419/zpn3s949