In a randomized, double-blind, sham-controlled study of infants with spinal muscular atrophy (SMA) type 1, nusinersen treatment led to significant improvement in motor milestones and survival without significant adverse reactions. A safety trial of gene therapy for SMA type 1 demonstrated prolonged ventilator-free survival and improved motor milestones with minimal side effects.
This study comparing 20 individuals who received MRI-guided focused ultrasound thalamotomy with seven individuals who received sham treatment showed improvements in Parkinson’s disease tremor. Side effects were similar to prior studies of this technology for essential tremor, with ataxia and limb/orofacial paresthesias being most common.
In a Phase II, single-blind, randomized, multicenter trial, the use of intraparenchymal brain tissue oxygenation monitoring reduced brain tissue hypoxia in patients with severe traumatic brain injury.
Activation of the immune system has been recognized in patients with amyotrophic lateral sclerosis, and immune activation may influence the course of the disease and the speed of progression.
Anti-programmed death 1 antibodies (checkpoint inhibitors) have become a mainstay in the treatment of many types of cancers, and now are known to cause frequent neuromuscular adverse effects that can cause severe disability or death if not recognized or treated promptly.
