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Breakthrough treatment for kids with NF1 muscle weakness

By December 8, 2021January 17th, 2024NF1, Science & Research

We are excited to share an exciting breakthrough treatment from a research study co-led by CTF-funded researcher Aaron Schindeler, Ph.D. of The Children’s Hospital at Westmead, Australia. This treatment has been successfully trialed in a study and has the potential to help children with NF1 who complain of muscle weakness and fatigue. The researchers found that L-carnitine, a supplement used by athletes to prevent muscle fatigue, can considerably improve muscle function in children with NF1. The work was supported by the Children’s Tumor Foundation.

Read about the impact this new treatment has had on the participants living with NF1-related muscle weakness in the 12-week trial here.

CTF has contributed to both the preclinical and clinical studies that led to this incredible and very promising result, most recently in the Drug Discovery Registered Reports program, and the CTF-funded preclinical study, which you can read about here. The CTF Clinical Research Award scheme partially funded the clinical study back in 2018 which led to this publication.