Author Archives: Lila Levinson, PhD

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…

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…

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…

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…

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…

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…

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…

A real-world study of Spinraza (nusinersen) use in Switzerland among individuals with spinal muscular atrophy (SMA) found that, while patients tended to experience motor function benefits mostly in their first year of treatment, many…

Most adults and adolescents with spinal muscular atrophy (SMA) experience intermittent pain, with more than half having pain episodes at least once a week, a study reports. This pain was generally mild to moderate and affected the…

The European Commission (EC) has approved a tablet formulation of spinal muscular atrophy (SMA) treatment Evrysdi (risdiplam). “The new Evrysdi tablet with its flexible administration represents progress toward more versatile SMA disease management,” Levi Garraway, MD, PhD, chief…