News

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Apic Bio‘s APB-102, an investigational gene therapy aiming to treat familial amyotrophic lateral sclerosis (ALS) associated with mutations in the superoxide dismutase 1 (SOD1) gene. “This orphan drug designation represents an important…

While rehabilitation programs that include exercise are helpful to amyotrophic lateral sclerosis (ALS) patients, no extra benefit was seen by raising the number of weekly sessions from a standard two to five, a small study from Italy reports. The study, “High‐frequency motor rehabilitation in amyotrophic lateral sclerosis: a randomized…

The gut microbiome — the natural collection of microorganisms living in our guts —  might influence the progression of amyotrophic lateral sclerosis (ALS), recent research suggests. Specifically, researchers found that a metabolite produced by the Akkermansia muciniphila bacteria, called nicotinamide, slowed disease progression and prolonged survival in an ALS mouse…

Concerns over an active pharmaceutical ingredient used in a 2017 study of Nurtec (riluzole) as a bioequivalent medicine were voiced by the U.S. Food and Drug Administration (FDA) in its response to a request for the treatment’s approval for  amyotrophic lateral sclerosis (ALS), its developer, Biohaven Pharmaceuticals,…

Patient enrollment is now complete, ahead of schedule, for the Phase 3 ORARIALS-01 clinical trial assessing the effectiveness of Orphazyme’s investigational therapy arimoclomol in the treatment of amyotrophic lateral sclerosis (ALS). “The completion of enrollment for Orphazyme’s pivotal ALS trial is another great milestone for our clinical development program. We are confident that…