We’re excited to share some encouraging news on the front lines of Nemaline Myopathy research. Dr. Afrooz Rashnonejad and her team, funded by the support of AFBS, have been making strides in developing a potential gene therapy for NEM3, a form of Nemaline Myopathy caused by mutations in the ACTA1 gene. Nemaline Myopathy Gene Therapy:…
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On the Blog
Research Brief: Promising Updates in Nemaline Myopathy Gene Therapy and Drug Repurposing Development
Dr. Vandana Gupta and her team at Brigham & Women’s Hospital, with funding from A Foundation Building Strength (AFBS), are making exciting progress in the quest to find treatments for Nemaline Myopathy (NM). This rare neuromuscular disease affects muscle function and can significantly impact quality of life. Here’s a quick update on their recent research…
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