"The strongest, most resilient babies," the Little Mix singer said of her daughters Ocean Jade and Story Monroe ...

Jesy Nelson hasn’t given up hope that her twin daughters, Ocean Jade and Story Monroe, will overcome a muscle disease they were diagnosed with a month ago. The former Little Mix singer was recently a ...

A Prescription Drug User Fee Act target date of September 22, 2025 has been set for the application. The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License ...

A mum has been left "overwhelmed" after a fundraiser raised more than £5,000 to help her purchase a spinal brace for her son.

Findings from DEVOTE support clinical benefits of the high-dose regimen of nusinersen (50 mg and 28 mg) in both ...

Infantile spinal muscular atrophy (SMA) is a neuromuscular disorder that affects the nerve cells in the spinal cord. It causes muscles to weaken and limits muscle movement. SMA is a rare condition.

"Managing pain in patients with SMA begins with the recognition that there are predictable etiologies leading to pain in SMA and investigating them during each medical visit is important to implement ...

Biogen recorded declining sales for Spinraza in each of the four quarters of 2025, but the Massachusetts drugmaker sees ...

People with spinal muscle atrophy (SMA), an inherited neuromuscular disease, usually experience muscle weakness that impacts movement. New research suggests that electrical spinal cord stimulation ...

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 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 ...