—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 ...

What Is Itvisma, and Why Does It Matter? Itvisma (onasemnogene abeparvovec-brve) is a gene therapy that the FDA has approved to treat spinal muscular atrophy (SMA) in adults, teens, and children 2 or ...

In a first-of-its-kind study, researchers compared the efficacy of preventative therapy for spinal muscular atrophy (SMA) between two well-matched study groups, using either gene therapy (onasemnogene ...

Longitudinal data on skeletal muscle growth in children are scarce, so little is known about developmental trajectories of skeletal muscle size, the relative timing of muscle and skeletal growth and ...

In recent years, the advent of three disease-modifying therapies (DMTs) for Spinal Muscular Atrophy, a progressive neuromuscular condition, has altered the natural history of the disease and led to ...

Duchenne muscular dystrophy (DMD) is an inherited muscle disorder that causes progressive breakdown of muscle tissue. Symptoms typically begin in early childhood, and most parents and caregivers ...