The tiny zebrafish is helping researchers rapidly determine whether a newborn's genetic mutation is likely to cause spinal ...

Editor’s note: This is an automatically generated transcript. Please notify [email protected] if there are concerns regarding accuracy of the transcription. So most recently, there has been the ...

"Decreasing AMMO [active maximal mouth opening] over time is seen most often in patients with SMA type 2 with an estimated reduction of almost 1 mm per year...At this point, we propose that ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

"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 ...

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 ...

Injections for spinal muscular atrophy (SMA) refer to two treatments that the Food and Drug Administration (FDA) has approved for SMA: nusinersen (Spinraza) and onasemnogene abeparovec-xioi (Zolgensma ...

An insurance company has denied newborn twins a one-time genetic treatment that could stop the progression of their spinal muscular atrophy — otherwise they are likely to die before their second ...

Editor’s note: This is an automatically generated transcript. Please notify [email protected] if there are concerns regarding accuracy of the transcription. So for spinal muscular atrophy, we have ...

Panelists discuss how spinal muscular atrophy is an autosomal recessive genetic disease affecting motor neurons with 3 currently approved disease-modifying therapies that restore SMN protein ...

Health Editor Martin Bagot explains how inspirational families he spoke to for the Mirror’s spinal muscular atrophy (SMA) ...

SCHAUMBURG, Ill., Nov. 26, 2025 /PRNewswire/ -- Cure SMA, the leading nonprofit organization dedicated to supporting those impacted by spinal muscular atrophy (SMA), welcomes the FDA approval of ...