New York, Dec. 18, 2024 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) and AFM-Téléthon (the French muscular dystrophy association) 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). These collaborative efforts represent the MDA’s commitment to funding innovative research with the potential to transform lives.
“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.”
Michele Brischigliaro, PhD, from the Miller School of Medicine at the University of Miami, will receive $210,000 over a three-year period (10/1/2024–9/30/2027) 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 $300,000 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.
“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-Téléthon. “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.”
The Muscular Dystrophy Association continues to lead the way in neuromuscular disease research, fostering collaboration with key global partners like AFM-Téléthon to advance scientific breakthroughs. For more information about the MDA’s research initiatives, visit www.mda.org.
For press inquiries please email press@mdausa.org.
About Muscular Dystrophy Association
Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and over 300 related neuromuscular conditions. For nearly 75 years, MDA has led the way in accelerating research, advancing care, and advocating for the support of our families. MDA's mission is to empower the people we serve to live longer, more independent lives. To learn more visit mda.org and follow MDA on Instagram, Facebook, X, Threads, TikTok, LinkedIn, and YouTube.
About AFM-Téléthon
The French Muscular Dystrophy Association (AFM) federates patients with neuromuscular diseases and their families. Thanks to donations from France's annual Telethon, the AFM-Telethon has become a major player in biomedical research for rare diseases in France and worldwide. It currently funds about 350 research programs and 40 clinical trials in different genetic diseases affecting the eye, blood, brain, immune system, motor neurons and muscles... The AFM-Telethon has also created three research and development institutes dedicated to gene therapy, stem cells and myology. To learn more visit www.afm-telethon.fr/en.
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