FDA Approves Golodirsen for Duchenne Muscular Dystrophy
The FDA has granted accelerated approval to Vyondys 53 (golodirsen; Sarepta Therapeutics) injection for the treatment of Duchenne muscular dystrophy.
Neuromuscular Disorders
Risdiplam Gets Priority Review for Spinal Muscular Atrophy
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.
Risdiplam for Spinal Muscular Atrophy Meets Primary End Point in Motor Function
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.
Spironolactone Noninferior in Duchenne Muscular Dystrophy
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.
Trials Evaluating Intrathecal Gene Treatment for Spinal Muscular Atrophy Placed on Partial Hold
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.
Antiviral Antibodies Identified in CSF of Patients With Acute Flaccid Myelitis
Enterovirus (EV)-specific antibodies have been identified in the cerebrospinal fluid (CSF) of pediatric cases of acute flaccid myelitis (AFM) versus controls.
Muscle Involvement Seen in Dermatomyositis With and Without Anti-Mi2 Autoantibodies
Muscle disease is more prominent among patients with dermatomyositis and anti-Mi2 autoantibodies compared with in those who have anti-Mi2-negative dermatomyositis.
Vamorolone Shows Dose Related Improvement in Young Patients With Duchene Muscular Dystrophy
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.
Nusinersen Delays Respiratory Intervention in Presymptomatic Infants With Spinal Muscular Atrophy
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.
Baseline Findings Predict Magnitude of Change in Patients With Type 2 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.
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