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.
The Food and Drug Administration (FDA) has approved Myobloc (rimabotulinumtoxinB injection; US WorldMeds) for the treatment of chronic sialorrhea in adults.
The FDA has issued a complete response letter to Sarepta Therapeutics regarding the NDA for golodirsen, an investigational treatment for patients with Duchenne muscular dystrophy with a confirmed mutation amenable to exon 53 skipping.
A new rapid turnaround genetic test for spinal muscular atrophy, found to reduce time to results from 21 days to 4 days, will be offered at no charge to qualified individuals in the United States as part of the SMA Identified program.
This case study illustrates that severe proximal myopathy can be secondary to hypothyroidism. It also emphasizes the importance of measuring thyroid function in patients with proximal weakness, myalgia, and a significantly raised creatine kinase.
According to the Food and Drug Administration (FDA), AveXis Inc, the manufacturer of the gene therapy Zolgensma, was aware of an issue related to “data manipulation” prior to approval, but did not inform the Agency until after the product was approved.
The Food and Drug Administration (FDA) has approved Ruzurgi (amifampridine; Jacobus Pharmaceutical) for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in patients 6 to <17 years of age.
Preliminary findings showed the decrease in botulinum toxin type A therapeutic effect over time warrants further study.
Cigarette smoking is associated with more severe multiple sclerosis disease course with lower lifetime cigarette smoking load and smoking cessation having no significant effect on disease severity.
Guidance has been updated for the health supervision of children with neurofibromatosis type 1.
