CUMBERLAND COUNTY, Pa. — Nicole Moore of Cumberland County is accomplishing goals she previously thought were impossible. Thanks to a revolutionary at-home treatment, she’s becoming more independent ...

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 ...

When we think about genetic diseases, the ones that usually come to mind are cystic fibrosis, Fragile X and, of course, Tay-Sachs disease. One disease that many people are not aware of is spinal ...

MADERA COUNTY, Calif. (KFSN) -- Like most one-year-olds, Damaris Perez spends a lot of time playing in her crib. The average person wouldn't know this baby has limited time left on earth. "It's been ...

Add Yahoo as a preferred source to see more of our stories on Google. This image from video provided by UPMC and University of Pittsburgh Health Sciences shows Doug McCullough, who has spinal muscular ...

I feel like my whole life has done a 360,” says Jesy Nelson in the final five minutes of Jesy Nelson: Life After Little Mix (Prime Video), the six-part documentary charting her life since leaving ...

Young internet sensation Neilah, living with spinal muscular atrophy, has passed away. Her mother, Ruth Wambui, shares their ...

(WNDU) - Imagine your baby being born healthy, and then within months, they start to lose the ability to move their arms and legs, fingers, and toes. This is what happens to infants born with spinal ...

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 ...

"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 ...