News

At-home Music Therapy Program May Help Bulbar-onset Patients

An at-home music therapy program aimed at improving speech, swallowing, and respiration in people with amyotrophic lateral sclerosis (ALS) was well-tolerated and feasible, according to a small pilot study. While the pilot study (NCT03604822) was not designed to test the program’s effectiveness, collected data suggest that the music…

FDA Grants Orphan Drug Status to TQS-168 for ALS Treatment

The U.S. Food and Drug Administration has granted orphan drug designation to Tranquis Therapeutics‘ experimental therapy TQS-168 for treating amyotrophic lateral sclerosis (ALS). Orphan drug status is given to experimental therapies with promise for treating rare diseases — those affecting fewer than 200,000 people in the U.S.

ILB Safe, Shows Potential to Slow ALS Progression in Small Phase 2 Trial

TikoMed reported that ILB, its investigational therapy to protect nerve cells in amyotrophic lateral sclerosis (ALS), was well-tolerated by patients, and led to improvements in mobility and disease severity while reducing spasticity scores in a small open-label clinical trial. “These early results from this ALS-study are exciting as ILB provided beneficial…

SBT-272, in Phase 1 Trial, Continues to Protect Motor Neurons in Mice

Stealth BioTherapeutics’ investigational therapy SBT-272 eased inflammation and upper motor neuron degeneration in a mouse model of amyotrophic lateral sclerosis (ALS) associated with the accumulation of toxic TDP-43 protein clumps, the company announced. Motor neurons are nerve cells that control voluntary movement and progressively die in people with ALS.

B12 Slows Functional Decline in Early-stage ALS, Data Show

Treatment with ultrahigh-dose methylcobalamin — the physiologically active form of vitamin B12 — effectively slowed functional decline in people with early-stage amyotrophic lateral sclerosis (ALS) and moderate progression, data from a small Phase 3 trial in Japan show. The findings confirm previous results from a Phase 2/3 trial and…