April 2019

Currently, there are no disease-modifying medications for Huntington disease and few interventions available to effectively alleviate symptoms, while the lack of indicators of pathobiological processes at the molecular level has limited the ability to prove efficacy and safety in clinical trials.

Although highly efficacious DMTs have improved outcomes for a growing number of patients, the majority of people with MS are still subjected to the day-to-day consequences of the disease’s symptoms, which often have a more debilitating effect on the patient than the disease itself.

The investigational candidate is the subject of the 2-part phase 2/3 SUNFISH trial in young adult and pediatric patients with type 2 and type 3 SMA.

NeurologyLive spoke with the director of the Cleveland Clinic Lou Ruvo Center for Brain Health to learn why, despite hundreds of failed trials and therapies, there is growing excitement for what's to come.

Research from the past decade suggests that sigma-1 receptors and their encoding gene, SIGMAR1, together act as a therapeutic target for patients with dementia.

The approval of nusinersen in 2016 to treat spinal muscular atrophy marked an important milestone in efforts to treat the disease, but even more breakthroughs could be coming soon.