Modifications that help certain genetic information travel to specific parts of nerve cells can ease motor symptoms and help normalize communication between nerves and muscles in a mouse model of spinal muscular atrophy (SMA), a study reported. SMA disrupts this nerve-muscle communication, which occurs at sites called neuromuscular junctions…
Using a specially designed back brace in addition to taking part in physical therapy may work best to reduce abnormal spine curvature, or scoliosis, in children with spinal muscular atrophy (SMA) type 1, a new study from Turkey reports. During eight weeks of daily physical therapy, which…
A technique to estimate the health of nerve cells that control movement may help predict how strongly some individuals will respond to the spinal muscular atrophy (SMA) gene therapy Zolgensma (onasemnogene abeparvovec-xioi), a study reports. This metric, called the cumulative motor index (CMI), assesses four major movement-related nerves.
Biogen has acquired Alcyone Therapeutics and will continue to develop ThecaFlex DRx, a device intended to ease patient experience and accessibility when delivering certain medicines into the spinal canal. The ThecaFlex DRx System is initially being evaluated with Spinraza (nusinersen) in people with spinal muscular atrophy (SMA),…
Measuring the levels of the survival motor neuron (SMN) protein in blood cells may help track spinal muscular atrophy (SMA) patients’ response to Evrysdi (risdiplam), a study from Japan found. Patients taking the drug showed a significant increase in blood levels of SMN after one month of therapy. SMN…
A man with type 3 spinal muscular atrophy (SMA) and no history of diabetes developed diabetic ketoacidosis, a severe diabetes complication, which researchers said may be linked to metabolic changes. SMA can cause major loss of muscle, which plays a key role in processing sugars and fats. Muscle loss…
An infant with spinal muscular atrophy (SMA) type 1 and neutropenia was successfully treated with with Zolgensma (onasemnogene abeparvovec-xioi), according to a case report. Neutropenia, which refers to low levels of immune cells called neutrophils, increases the risk for infections. However, the child “tolerated the treatment well and,…
Most parents and potential parents in the U.K. support screening newborns for spinal muscular atrophy (SMA) as part of the National Health Service (NHS) Newborn Screening Programme, according to a survey developed and funded by Novartis. “We believe that this government has an opportunity to make newborn screening…
Molecular markers in the liquid that surrounds the brain and spinal cord, called the cerebrospinal fluid (CSF), may help predict how well people with type 2 or 3 spinal muscular atrophy (SMA) respond to Spinraza (nusinersen), a study reports. The study’s researchers found that certain CSF molecules involved…
A group of questionnaires called PROfuture can meaningfully measure a variety of symptoms of spinal muscular atrophy (SMA) that impact patients, a study reported. “The PROfuture questionnaires, developed by patients for patients in the current era of new treatments for SMA, can help to better characterize the impact of…
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