AFM-Telethon and Muscular Dystrophy Association Announce Research Grant Awards Advancing Treatments for Mitochondrial Myopathies and ALS
AFM-Téléthon and The Muscular Dystrophy Association (MDA) are proud to announce the awarding of two cutting-edge research grants to accelerate the development of treatments for mitochondrial myopathies and amyotrophic lateral sclerosis (ALS).
“This partnership with MDA is an essential step to build a strong collaboration between our two associations, through the support of these two promising projects,” said Jean-François Briand, PhD, Operations and Scientific Innovation Director, AFM-Telethon. “They both explore different RNA involvement in rare diseases that will open the way to new therapeutic development not only for these diseases but also for other ones.”
Two innovative research projects supported :
- Michele Brischigliaro, PhD, from the Miller School of Medicine at the University of Miami, will receive a grant over a three-year period for his groundbreaking research titled "Aberrant mt-mRNA folding as mediator of mitochondrial encephalomyopathies." His work will explore how defects in mitochondrial gene expression contribute to the development of mitochondrial diseases, which disproportionately affect children and lead to severe neurological and cardiac symptoms. The research focuses on two critical genes, ATP6 and ATP8, and how errors in their function lead to disease, with the ultimate goal of identifying new therapeutic approaches.
- Eran Hornstein, MD, PhD, from the Weizmann Institute of Science, will be awarded for a three-year research project (10/1/2024–9/30/2027) titled "microRNA as biomarkers for phenoconversion in pre-familial ALS." His research seeks to identify early biological markers that can signal the onset of ALS symptoms. By identifying and studying microRNAs as potential biomarkers, Dr. Hornstein aims to uncover critical pathways that could pave the way for earlier diagnosis and new treatment strategies for ALS, a devastating neurodegenerative disease.
“We are pleased to partner with AFM-Téléthon to support these two excellent researchers whose work holds such promise for the neuromuscular disease community,” said Sharon Hesterlee, PhD, EVP and Chief Research Officer, Muscular Dystrophy Association.“These grants will enable us to take crucial steps forward in understanding the underlying mechanisms of these diseases, leading to new and innovative treatments that can significantly improve patients’ lives.”
AFM-Telethon continues to lead the way in neuromuscular disease research, fostering collaboration with key global partners like The Muscular Dystrophy Association to advance scientific breakthroughs.