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.
In a head-to-head study of mineralocorticoid receptor antagonists, spironolactone was found to be noninferior to eplerenone for slowing the progression of heart damage in boys with Duchenne muscular dystrophy.
The Food and Drug Administration (FDA) has placed a partial hold on clinical trials for the intrathecal administration of AVXS-101 (AveXis, a Novartis Company) in patients with spinal muscular atrophy (SMA) Type 2.
Enterovirus (EV)-specific antibodies have been identified in the cerebrospinal fluid (CSF) of pediatric cases of acute flaccid myelitis (AFM) versus controls.
Muscle disease is more prominent among patients with dermatomyositis and anti-Mi2 autoantibodies compared with in those who have anti-Mi2-negative dermatomyositis.
Daily vamorolone treatment for Duchenne muscular dystrophy suggested efficacy at doses of 2.0 and 6.0 mg/kg/d in an exploratory 24-week open-label study.
Positive data from phase 2 study of Spinraza for preventing or delaying the need for respiratory intervention or death in infants with genetically diagnosed and presymptomatic spinal muscular atrophy.
In patients with type 2 spinal muscular atrophy (SMA), the severity of functional impairment at baseline can assist in predicting the magnitude of changes over time, as well as the onset of scoliosis and the need for noninvasive ventilation.
The FDA has granted Fast Track designation to ANX005 for the treatment of Guillain-Barré Syndrome, an autoimmune disease that can lead to widespread peripheral nerve damage and paralysis.
The Food and Drug Administration has granted Orphan Drug designation to zilucoplan for the treatment of generalized myasthenia gravis and other rare, tissue-based complement-mediated diseases.
