SOL-257, an experimental gene therapy developed by SOLA Biosciences, delayed disease progression and prolonged the lifespan of a mouse model of amyotrophic lateral sclerosis (ALS). The gene therapy is designed to reduce the abnormal TDP-43 protein that forms toxic clumps in the cells of ALS patients. “Targeting only…

A Phase 3 clinical trial evaluating Orphazyme’s investigational oral therapy arimoclomol for amyotrophic lateral sclerosis (ALS) has failed to meet its primary and key secondary goals. While no safety issues were reported in the trial (NCT03491462), arimoclomol did not significantly extend patients’ lives nor…

Jan Veldink, a neurologist from the Netherlands whose work in the genetics of amyotrophic lateral sclerosis (ALS) has led to important discoveries about disease risk, won the 2021 Sheila Essey Award, according to a blog post  from the ALS Association. The $50,000 award was presented at the recent…

A combination of guanabenz — an approved therapy for high blood pressure — plus riluzole slowed disease progression in adults with early amyotrophic lateral sclerosis (ALS), particularly those with bulbar onset disease, a form of ALS, according to data from a Phase 2 clinical trial. Despite these promising benefits, the…

“Everybody’s talkin’ at me, Can’t hear a word they’re sayin’, Only the echoes of my mind.” – Fred Neil ALS has rendered me a minimalist creature of habit. My weekday routine includes the intersection of paranoid caution, practiced routine,…

Registration is now open for fundraising events at four championship golf courses, whose fees will be used to fund research, care, and advocacy for neuromuscular disorders such as amyotrophic lateral sclerosis (ALS) and muscular dystrophy. According to the…

Editor’s note: The ALS News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read the latest stories from the conference. Amyotrophic lateral sclerosis (ALS) patients require walking aids, ventilation support, and/or speech generation devices about 1.5 years after starting treatment with…

Speech and oral therapy were added to covered services available by telemedicine for people with amyotrophic lateral sclerosis (ALS), which Medicare previously recognized only as in-person visits, during the COVID-19 pandemic. Medicare will now reimburse — at least through December 2021 — evaluations and treatments given by telemedicine…

Editor’s note: The ALS News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read the latest stories from the conference. A new clinical trial will seek to determine the optimal timing to begin treatment with the investigational medication tofersen in people…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…