STRENGTHENING the global effort to CURE neuromuscular disease

Children born with congenital muscular dystrophies face progressive muscle weakness from birth, often leading to severe disability and shortened lifespans. While promising treatments are entering clinical trials, researchers have struggled with a critical challenge: how to accurately measure whether these treatments are working.

Rotem Orbach, MD, from the National Institutes of Health, is a 2025 American Neuromuscular Foundation Development Grant recipient who is pioneering a solution using multispectral optoacoustic tomography (MSOT), a revolutionary imaging technology that could change how doctors monitor muscle diseases. Unlike current methods that rely on a child's ability to perform tasks or require uncomfortable procedures like MRI scans with sedation, MSOT is completely non-invasive, radiation-free, and can be used right at the bedside.

The technology works by visualizing changes in muscle composition, such as scarring and fat buildup, providing objective measurements of disease progression. Dr. Orbach's research focuses on the two most common forms of congenital muscular dystrophy, comparing affected patients to healthy individuals over time.

If successful, MSOT could revolutionize clinical trials by making them faster, more accurate, and more effective at bringing new treatments to patients. The technology's safety and ease of use could eventually make it a routine tool in neuromuscular clinics, helping doctors make better treatment decisions and giving families clearer information about their child's condition and response to therapy.

Research like this is made possible thanks to the generous support of donors. Support the future of neuromuscular medicine with a with a donation today.