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...
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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...
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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...
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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...
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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...
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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...
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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...
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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...
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FDA Grants Rare Pediatric Disease Designation to SRK-015 for SMA

The U.S. Food and Drug Administration (FDA) has granted the designation of rare pediatric disease to SRK-015, an investigational treatment for spinal muscular atrophy (SMA), its developer, Scholar Rock, announced. This designation is given to therapies that are...
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Nerve-muscle Communication Molecule May Be SBMA Therapeutic Target, Study Says

Increasing the levels of brain-derived neurotrophic factor (BDNF) — a molecule involved in the communication between nerve cells and muscle — specifically in muscle delayed symptom onset and slowed disease progression in a mouse model of spinal and bulbar muscular...
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Nerve-muscle Communication Molecule May Be SBMA Therapeutic Target, Study Says

Increasing the levels of brain-derived neurotrophic factor (BDNF) — a molecule involved in the communication between nerve cells and muscle — specifically in muscle delayed symptom onset and slowed disease progression in a mouse model of spinal and bulbar muscular...

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Lung Health Declines in Step With Motor Loss in Types 2 and 3, Study Finds

Lung Health Declines in Step With Motor Loss in Types 2 and 3, Study Finds

Nov 24, 2020

Declines in lung function are steeper and quicker in children with spinal muscular atrophy (SMA) type 2 than non-ambulatory children with SMA type 3, matching changes seen in motor abilities in these two patient groups over time, an eight-year study of untreated...

SMA Patient Columns

My Experience with the JACO Robotic Arm

Throughout my life, I have required assistance with the most basic tasks. Things like scratching my head, getting myself a drink, or picking something up off the floor are just a few items on a never-ending list of impossibilities. Fortunately, there is now technology...
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The Amino Acid Diet and Its Benefits

Nutrition has always been a challenge for my daughter, Ella. And, in turn, it is a challenge for my wife and me. Due to Ella's disease, she has an increased amount of difficulty digesting long-chain fats (meats, cheeses, etc.) and she also has a hard time maintaining...
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