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Gene therapies have extended survival in muscular dystrophy, requiring improved care transitions from pediatric to adult systems. Current systems lack adequate staffing, specialists, and communication ...

Ann & Robert H. Lurie Children’s Hospital of Chicago and the Muscular Dystrophy Association have announced a new clinical partnership to enhance care for children with neuromuscular diseases. Under ...

In research published in Developmental Medicine & Child Neurology, investigators have developed a brief, reliable, and valid ...

—A new qualitative participatory study explored how powered mobility (PM), in the form of modified ride-on cars, impacts children with spinal muscular atrophy type 1 (SMA1) and their families. Here ...

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today new 5-year data confirming the sustained efficacy and safety profile ...

Researchers evaluated oxygen saturation and arterial CO2 variables to screen for sleep disordered breathing in children with SMA type 1-3. Of the measures evaluated, only an oxygen desaturation index ...

Duchenne muscular dystrophy (DMD) results from changes in the DMD gene. Children may inherit DMD gene changes from their parents or have spontaneous gene changes. The DMD gene tells the body how to ...

Each week, this series shares MarionMade! stories of our many wonderful people, places, products and programs in the greater Marion community. To read more positive stories of Marion, or to share some ...

WASHINGTON, June 12, 2025 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), is pleased to announce the ...