News

Muscle ultrasound may help to monitor SMA progression: Study

Muscle abnormalities observed with ultrasound imaging correlated with motor function in people with spinal muscular atrophy (SMA) in a recent study. While the findings varied somewhat by muscle group and SMA type, ultrasound data generally indicated SMA patients exhibited muscle atrophy and signs of fat and connective tissue…

Study finds few cognitive changes after Spinraza treatment

More than a year of treatment with Spinraza (nusinersen) did not result in clinically meaningful changes in cognition for adults with spinal muscular atrophy (SMA), according to a recent study. While patients did not exhibit widespread cognitive deficits, abnormal performance in certain domains was observed. Scores across most…

NIH awards The Jackson Laboratory over $22M for gene editing research

Scientists at The Jackson Laboratory — a U.S.-based biomedical research nonprofit — were granted $22.8 million by the National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health (NIH), for a novel method to develop gene editing therapies for neurological disorders, including spinal…