Bulbar muscles, those of the face and throat, are affected in spinal muscular atrophy (SMA), and researchers in Germany found that young children with SMA cannot open their mouths as well as age-matched infants and toddlers without this disease. “There is a growing interest in the swallowing and the…
Problems in how dopamine and other chemical messengers work in nerve cells can cause issues with posture in spinal muscular atrophy (SMA), according to a new study in mice that also suggests that levodopa therapy — which works by increasing the levels of dopamine — could help ease such…
Measuring the amount of gray matter in the spinal cord could help doctors track spinal muscular atrophy (SMA) over time and see how well treatments are working, according to a study that found shrinking gray matter was tied to weaker muscles and worse motor function. More research is needed…
Regulators in the U.S. and the European Union have agreed to review Biogen’s applications seeking approval of a higher dose of Spinraza (nusinersen) for all types of spinal muscular atrophy (SMA) in individuals of all ages — a regimen the therapy’s developer says may provide more effective treatment…
Gratitude and support from family and healthcare staff can help people with spinal muscular atrophy (SMA) feel more positive, stay motivated, and plan their lives better, a study found. Programs that focus on gratitude could help patients improve their mental health and help them cope with their disease more…
Slightly more than half of spinal muscular atrophy (SMA) patients in Australia who gave feedback for a study on how well they thought their treatments worked did not notice any improvement. The researchers said the results suggest a need for better, more comprehensive healthcare. More children than adults with…
Scholar Rock’s apitegromab led to motor function improvements over a year of treatment in young adults and children with spinal muscular atrophy (SMA) type 2 or 3 who could sit independently and were on stable standard of care, but unable to walk. That’s according to…
SMA Europe has released a new documentary titled “One Community. Shared Dreams.” as part of an awareness initiative aimed at highlighting the importance of interdisciplinary collaboration among researchers to advance the understanding of spinal muscular atrophy (SMA). The documentary shares the personal stories of people around the…
A baby’s respiratory failure and need for mechanical ventilation were traced to spinal muscular atrophy with respiratory distress type 1 (SMARD1), a very rare disease, with one of her two IGHMBP2 gene mutations never before reported. “This case expanded knowledge on the genetic profile of SMARD1 and it provides…
Spinal muscular atrophy (SMA) may involve changes that occur before birth, during an embryo’s development in the womb, and not just after an infant is born, a study in cell structures suggests. This could mean that combining available disease-modifying treatments with medications that address early changes could help better…
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