A Physical Therapist’s Perspective On a New Treatment for SMA in Patients 2 Months and Older Sponsored Post
As key team members in the management of spinal muscular atrophy (SMA),1physical therapists need to understand how disease-modifying therapies may impact patients with SMA and how to best assess these patients over time. Evrysdi (risdiplam) is a new disease-modifying...
Molecule Similar to Evrysdi But Possibly Safe at Higher Doses Identified
A newly identified small molecule able to cross into the brain — named TEC-1 — significantly eased disease severity and prolonged survival in a mouse model of spinal muscular atrophy (SMA), a study from Japan reported. TEC-1 was reported to work in ways similar to...
Massage and Stretching Techniques For SMA Patients
During physical therapy, there are many different techniques that can be utilized to maximize the patient’s care. Some of these can include different manual techniques such as soft tissue massage, edema reduction massage, and manual stretching to increase the comfort...
Muscle-directed Therapy SRK-015 Improves Motor Function in SMA Types 2, 3, Data Show
SRK-015, Scholar Rock’s muscle-targeted therapy, safely and effectively improved motor function in children and young adults with spinal muscular atrophy (SMA) types 2 and 3, six-month interim data from a Phase 2 trial show. These benefits were more pronounced with...
SMA Type 3 Adults May Have Issues Regulating Component of Cell’s ‘Skeleton,’ Study Suggests
Adults with spinal muscular atrophy (SMA) type 3 have significantly lower levels of several molecules that help regulate actin, one of the major components of the cell’s cytoskeleton, according to a study. These findings, from blood and connective tissue cells, add to...
Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National...
Gene Therapy Reduces Disease Severity in SMA Mouse Model, Study Shows
Gene therapy that increased the production of a protein called DOK7 in a spinal muscular atrophy (SMA) mouse model was found to improve the neuromuscular junction structure, reduce muscle fiber loss, improve grip strength, and extend survival. These study findings...
Importance of Balance While Maneuvering on Uneven Surfaces For SMA Patients
Balance is typically a part of everyone’s treatment plan during physical therapy. Whether you have back pain or ankle pain, improving your balance carries over to an increase in functional mobility that will in turn improve your functional strength and increase your...
4-aminopyridine Fails to Improve Muscle Function in SMA Type 3 Patients, Small Trial Finds
Treatment with the molecule 4-aminopyridine (4-AP) failed to improve the motor function, endurance, and muscle strength of spinal muscular atrophy (SMA) type 3 patients who are able to walk, according to results from a small Phase 2/3 clinical trial. Findings from...HCP — SMA News Today
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