Children with spinal muscular atrophy (SMA) who were diagnosed through newborn screening and treated early had better long-term independence and daily functioning, especially when supported by multidisciplinary care at home and at school, according to a real-world study from Australia. “Multidisciplinary care is important to meet the changing and…
The U.S. Food and Drug Administration (FDA) is expected to decide in the coming months whether or not to approve apitegromab, an experimental muscle-strengthening agent designed to improve motor function in people with spinal muscular atrophy (SMA). Scholar Rock, the company developing apitegromab, resubmitted its application…
Discussion
Because I live with SMA, I value comfort and routine, which help to alleviate some of the hardship and stress that come with managing this disease. Since January, a new day nurse has been doing her best to provide these things for me. Her excellent care helps to protect…
Children with spinal muscular atrophy (SMA) who have two or fewer copies of the SMN2 gene — a feature typically associated with more severe disease — are more likely to start treatment early and reach key motor milestones if they are diagnosed through screening programs, a U.S. survey study suggests.
Discussion
I’m not one to turn down a compliment, but as a woman living with spinal muscular atrophy (SMA), I do not consider physical balance to be among my strongest skills. So, when my friend told me as we rode in the car one day that my balance was really…
A panel of experts has developed the first U.K. national consensus recommendations for preventing and managing hip problems in children with spinal muscular atrophy (SMA), aiming to support earlier monitoring and more consistent care in the era of disease-modifying treatments. The recommendations emphasize a more proactive and individualized approach…
Get regular updates to your inbox.
