The scientists at the University of California, San Francisco and Johns Hopkins University in Baltimore injected an antisense ...

A wearable robotic device called HAL improved walking ability for SMA patients who also received Spinraza (nusinersen), a ...

Richard Finkel, MD, a pediatric neurologist at St. Jude Children’s Research Hospital, was included in the Time100 Health list ...

Naim Alkhouri, MD, spoke to the potential for glucagon-like peptide-1 (GLP-1) receptor agonists to transform the treatment ...

Patients, families and staff gather for a group photo during the 2023 Dystroglycanopathies Patient & Family Conference (Photo courtesy of the University of Iowa Wellstone Center) Katherine Mathews, MD ...

(0:54) How does one time surgery for scoliosis in children with pediatric spinal muscular atrophy get a maximum benefit without impeding growth? Dr. Ashley E. Webb of the University of Texas ...