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 ...

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 ...

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 ...

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 ...

"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 ...

(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 ...

"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 ...

This is six year old Emma Nowll and like so many kids her age. She is *** prankster. You can also call her. She hates it when I try to help her so um the best thing I can do for her is to just teach ...

Spinal muscular atrophy (SMA) is a debilitating genetic condition that’s usually fatal by a few years of age. But an intriguing case study might demonstrate a simple new treatment, with a child ...

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 ...