The FDA has for spinal muscular atrophy (SMA) in pediatric and adult patients. Approval came about 4 months earlier than expected, and was based on an interim analysis of 82 of the 121 patients enrolled in the pivotal trial. The drug, developed by Ionis Pharmaceutical and marketed by Biogen, is likely to carry a price tag of about $250,000 per year. (FDA press release)
The day after Genetech announced a delay in the FDA's review of its multiple sclerosis candidate ocrelizumab (Ocrevus), full data from its three phase III trials -- ORATORIO for progressive MS and OPERA I and OPERA II in relapsing MS -- were published in the New England Journal of Medicine. (番茄社区)
Induced hypothermia for critically ill patients with convulsive status epilepticus. Patients had similar Glasgow Outcome Scale scores at 90 days whether they had the intervention added to standard care, or were treated with standard care alone, nor were there any differences in other secondary outcomes including mortality and progression to EEG-confirmed status epilepticus at 3 months. (New England Journal of Medicine)
Police in Derby, Conn., seized being transported by a truck driver from Arizona. (NBC Connecticut)
As the prevalence of neonatal abstinence syndrome has boomed with the opioid epidemic, effective management strategies have been developed -- but in terms of how the condition is defined and managed, researchers argue in the New England Journal of Medicine.
The FDA has warned that InBios/LabCorp's Zika test, ZIKV Detect, has a , and that physicians should confirm all results with the CDC.