News

Most of the infants and children with spinal muscular atrophy (SMA) in the RESPOND clinical trial who responded poorly to the gene therapy Zolgensma showed gains in motor function with Biogen’s Spinraza (nusinersen), according to early results. Prior preclinical studies have suggested Zolgensma may target…

A growing number of young children with spinal muscular atrophy (SMA) type 1 who started taking Evrysdi (risdiplam) as babies are gaining the ability to sit, stand, and walk, according to four-year data from the FIREFISH clinical trial. No new deaths have been reported since the study’s…

Add-on treatment with Scholar Rock‘s muscle-targeted therapy apitegromab continues to stabilize or improve motor function for young spinal muscular atrophy (SMA) types 2 and 3 patients who entered the study unable to walk, according to a three-year update from the TOPAZ clinical trial and its…

Researchers are calling for change in prenatal diagnostic procedures in Latvia after the first registered case of a newborn with spinal muscular atrophy (SMA) type 0 was reported in the country. “[It] is crucial to be able to detect this disease prenatally in order to provide the best…

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…

Many patients with spinal muscular atrophy (SMA) spend the majority of their time sitting in wheelchairs. Increased periods of sitting can lead to tailbone pain due to increased pressure on those areas. When these areas become stiff and painful, a little mobilization can lead to reduced pain and improved…

Electromyography (EMG)  is a test done with electrodes and/or needles inserted into or onto specific muscles to assess muscle activation at rest and during contraction. For Michael, my patient who has spinal muscular atrophy (SMA), we performed an EMG on different muscle groups to assess resting and activation…

Boosting the production of the growth factor protein NRG1-III improved motor nerve fiber development in the early stages in a mouse model of spinal muscular atrophy (SMA), a study showed. NRG1-III was unable to prevent nerve fibers from degenerating or improve motor function or survival in older mice,…

In adults with spinal muscular atrophy (SMA), treatment with Spinraza (nusinersen) in a clinical trial was tolerated well and led to improvements in arm function and self-rated health. These findings add to data from several studies that have examined the safety and efficacy of Spinraza in older…

Michael, a patient who has spinal muscular atrophy (SMA), has experienced reductions and increases in his lung capacity throughout his life due to his genetic disorder. This is a common occurrence in this population due to the disease’s progression on the muscles responsible for respiration. Just recently, this…