Muscle and Combo Therapies Likely Next Focus for SMA, Experts Say
Treatment combinations, including future muscle-targeted therapies, are part of what likely lies ahead for spinal muscular atrophy (SMA), according to experts in the SMA community. In separate interviews with SMA News Today, Richard Finkel and Laurent Servais,...
Researchers Discover Promising New Gene Therapy Approach for Spinal Muscular Atrophy
A new genetic approach to restore the expression of the gene that causes spinal muscular atrophy (SMA) in patients’ cells holds promise to effectively and permanently treat the disease, researchers suggest. The study, “Seamless genetic conversion of SMN2 to SMN1...
Spinraza-Zolgensma Combination Well-tolerated in Children with SMA Type 1, Study Shows
Combining Spinraza (nusinersen) with the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) is generally well-tolerated and sustains motor improvements in children with spinal muscular atrophy (SMA) type 1, according to a case series study. The data, which...
Edema Reduction Massage In SMA Patients
As we’ve discussed in previous articles, most patients with SMA sit in a wheelchair all day, therefore increasing the likelihood of experiencing increased swelling in their feet and ankles. For able-bodied individuals, when they walk, their legs are in a gravity...
Advancements in Wheelchair Options
As we have previously discussed in earlier articles, wheelchairs play a main role in the daily lives of many patients with SMA. Over the past ten years, wheelchairs have changed for the better, and now have many customizable options available that can very much...
Reality of SMA and Physical Therapy
Before meeting Michael, my patient with SMA, I had a lack in knowledge of what SMA really was, and what kind of goals were realistic to set for him to meet. I had no idea what my expectations should be regarding his progress and how to meet those expectations. I was...
Respiratory Weakness in SMA Most Pronounced in Childhood, Study Finds
Lung function in people with earlier-onset forms of spinal muscular atrophy (SMA) tends to decline gradually throughout childhood and stabilizes in early adulthood, according to data from a population-based natural history study. The study, “Natural history of lung...
Lung Ultrasound Reliable Option to Chest X-rays for Monitoring Children
Lung ultrasound is a safe and reliable bedside tool to detect atelectasis (partial lung collapse) early in children with neuromuscular diseases, including spinal muscular atrophy (SMA), a study suggests. This radiation-free method could be used to monitor lung health...
SBMA Seen to Affect Skeletal Muscles First, Not Motor Neurons
Although a disease of motor neurons, nerve cells of the spinal cord and brainstem, the earliest signs of spinal and bulbar muscular atrophy (SBMA) are in skeletal muscles, according to data from a mouse study. These findings suggest that changes in muscle strength and...HCP — SMA News Today
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