Kaitlin Batley, MD, a pediatric neurologist at Children’s Health and assistant professor at UT Southwestern Medical Center, highlights recent advancements and ongoing clinical trials in the spinal ...

Spinal muscular atrophy affects all the body's muscles. For a long time, it was considered a disease caused solely by the ...

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

The FDA has rejected a biologics license application for apitegromab to treat spinal muscular atrophy, according to manufacturer Scholar Rock. In a press release, Scholar Rock stated that the FDA’s ...

Panelists discuss how the current spinal muscular atrophy (SMA) treatment landscape includes 3 options: gene therapy (onasemnogene abeparvovec [Zolgensma]) for younger patients and 2 splice modifiers ...

The SMA treatment team also includes rehabilitation professionals such as respiratory, physical, occupational, and speech therapists. Rehabilitation treatment involves care related to positioning, ...

"Our preliminary findings show that German children with SMA, despite significant physical disability, have surprisingly good subjective HRQoL." – Erik Landfeldt, MSc, PhD Previous studies have found ...

Medical researchers have uncovered a novel mechanism that leads to motor neuron degeneration in spinal muscular atrophy (SMA). This discovery offers a new target for treatment that overcomes important ...

"The benefits of newborn screening identified in the study presented here encompass not only the achievement of motor milestones, but also a reduced need for ventilator support or tube feeding and a ...

Spinal muscular atrophy affects all the body’s muscles. For a long time, it was considered a disease caused solely by the ...

In a closely watched case, Roche suffered a setback as a court said it would permit a generic version of its rare disease ...