News

SMA News Today · #118: Aging and SMA   Long before Scholar Rock began its Phase 3 pivotal trial of apitegromab, the FDA had awarded Scholar Rock with…

Michael, my physical therapy patient with SMA, recently decided to have a subcutaneous port voluntarily implanted in his chest. As SMA patients age, and due to their disease, these patients may spend more time in the hospital than most, especially around the flu and pneumonia seasons. Due to having difficulty…

Levels of an immune protein called CHIT1 change over time in children with spinal muscular atrophy (SMA) who undergo treatment with Spinraza (nusinersen), a small study reports. The findings thus suggest that the protein’s levels — in the cerebrospinal fluid or CSF, the liquid surrounding the brain…

Treatment with Evrysdi (risdiplam) increased levels of SMN protein and stabilized motor function in a Phase 2 clinical trial that enrolled spinal muscular atrophy (SMA) patients who had previously been on other therapies, two-year data show. “These important data demonstrate the safety and efficacy of Evrysdi in…

The incidence of infantile spinal muscular atrophy (SMA) has increased in Japan since 2016 and this could be related to greater disease awareness among clinicians, earlier diagnoses, and the introduction of new treatments — although this relationship remains to be established, a study has found. On the large…

The gene therapy Zolgensma (onasemnogene abeparvovec-xioi), approved in the U.S. for very young children with spinal muscular atrophy (SMA), potentially could work for adults with the rare genetic disorder, a new study suggests. The study reports that few adults with SMA were found to have antibodies against…

Doctors need to be well-educated about options in the evolving treatment landscape to help patients with spinal muscular atrophy (SMA) make informed decisions, say neurologists in a panel discussion. The panel of four neurologists with experience in treating SMA patients was convened by Biogen, which markets…

Currently used clinical scales for men with spinal and bulbar muscular atrophy (SBMA) can be easily and reliably used remotely to monitor disease progression when patients are unable to visit a doctor’s office, according to a study in Italy. The study, “Reliable virtual clinical assessment in spino-bulbar…

For a boy with spinal muscular atrophy (SMA) type 2, wearing a robotic gait-training device at home led to mobility gains and enabled him to participate in new activities like games and sports, a case study has found. According to a nursing assessment, the training was well-tolerated, with…

Hip pain is about four times more common among children with spinal muscular atrophy (SMA) type 2 than among those with type 1 disease, according to a study on pediatric patients who were unable to walk. While nearly all children had evidence of hip joint problems on…