Patients with spinal muscular atrophy treated with a one-time infusion of onasemnogene abeparvovec-xioi continue to show clinically meaningful benefits even 5 years after dosing.
Several magnetic resonance biomarkers from the lower extremities may be predictive of future ambulatory function in patients with Duchenne muscular dystrophy.
Machine learning muscle magnetic resonance imaging-based tool may help physicians in the diagnostic process of muscular dystrophies.
Zilucoplan yielded rapid, clinically meaningful, and sustained improvement in patients with moderate to severe generalized myasthenia gravis.
Risdiplam is an investigational, orally administered liquid survival motor neuron-2 (SMN2) splicing modifier designed to increase and sustain SMN protein levels throughout the central nervous system and peripheral tissues of the body.
Skeletal maturation is significantly delayed in boys with glucocorticoid-treated Duchenne muscular dystrophy.
Clinical and laboratory characteristics of acute flaccid myelitis (AFM) cases differ in peak and nonpeak years.
The FDA has granted accelerated approval to Vyondys 53 (golodirsen; Sarepta Therapeutics) injection for the treatment of Duchenne muscular dystrophy.
Recently announced results from part 2 of the SUNFISH trial, which evaluated the safety and efficacy of risdiplam in patients with type 2 or 3 SMA aged 2 to 25 years, showed that the treatment met its primary end point.
Positive data announced from the pivotal part 2 of the SUNFISH trial that evaluated the safety and efficacy of risdiplam in adult and pediatric patients with Type 2 or Type 3 SMA.
