Forbes contributors publish independent expert analyses and insights. Spinal muscular atrophy affects the nerves that control muscle movement, leading to progressive weakening. As a result, infants ...

The Muscular Dystrophy Association (MDA) celebrates today’s announcement that the U.S. Food and Drug Administration (FDA) has approved a High Dose Regimen of SPINRAZA® (nusinersen) for the treatment ...

Please provide your email address to receive an email when new articles are posted on . At 5 years, 91% of pediatric patients treated with Evrysdi were alive, 81% without permanent ventilation. Data ...

The U.S. Food and Drug Administration has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of spinal muscular atrophy (SMA) in patients 2 years and older with confirmed mutation in ...

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 ...

AMES, Iowa – Ravindra Singh has spent years studying a gene that when missing or mutated causes spinal muscular atrophy (SMA), a deadly disease that’s among the most common genetic disorders in ...

Columbia researchers have discovered how a genetic defect leads to spinal muscular atrophy (SMA), a critical piece of information about the disease that neurologists have been seeking for decades. The ...

Please provide your email address to receive an email when new articles are posted on . Interim results also showed reduction of the neurodegenerative biomarker neurofilament light chain. Serious ...

RESEARCH TRIANGLE PARK, N.C. (WTVD) -- Three North Carolina children with a disease that could have taken their lives now have bright futures thanks to a study by the Research Triangle Institute. The ...

"Our findings suggest that patients with spinal muscular atrophy experience various types of respiratory events, which cannot be differentiated by oximetry. Thus, polysomnography combined with ...

"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 ...