Study using a new, child-friendly scale found patients up to age 2 had smaller mouth openings than healthy peers, risking ...

In surveys, parents of children with SMA and healthcare professionals in Sweden note the many ways the pandemic affected ...

Scholar Rock is asking the U.S. Food and Drug Administration (FDA) to approve apitegromab, its experimental add-on therapy designed to boost motor function in people with spinal muscular atrophy (SMA) ...