MDA’s Night of Hope in Atlanta to honor late ALS patient and family

ALS researcher Merit Cudkowicz to receive MDA legacy award

Renowned neurologist and amyotrophic lateral sclerosis (ALS) investigator Merit Cudkowicz, MD, of Massachusetts General Hospital has been named this year’s recipient of the Muscular Dystrophy Association (MDA) Legacy Award for Achievement in Clinical Research. The award, which recognizes outstanding accomplishments in neuromuscular research, will be presented March 20…

CNM-Au8 found to delay clinical worsening in ALS HEALEY trial

Treatment with CNM-Au8 significantly delayed clinical worsening in people with amyotrophic lateral sclerosis (ALS), according to new exploratory analyses from the therapy’s arm of the HEALEY ALS platform trial. The experimental therapy, from Clene Nanomedicine, had previously been found to reduce the risk of death by more…

FDA names RAG-17, targeting ALS gene mutations, an orphan drug

RAG-17, an investigational therapy from Ractigen Therapeutics to treat amyotrophic lateral sclerosis (ALS), has been designated an orphan drug by the U.S. Food and Drug Administration (FDA). Orphan drug status is given to medicines intended to treat life-threatening or chronically debilitating diseases that affect fewer than 200,000 individuals…

Slower ALS progression seen with long-term CNM-Au8 use in trial

Amyotrophic lateral sclerosis (ALS) patients treated earlier with CNM-Au8 in a clinical trial experienced a significantly slower disease progression than those who started treatment nine months later. These latest findings from the RESCUE-ALS Phase 2 trial (NCT04098406) and its open-label extension (OLE) study (NCT05299658) add to earlier reports that…