Parsing Science Podcast: Silencing an ALS Gene

How could a gene that causes one type of ALS be switched off? In episode 87, Tim Miller from the Washington University in St. Louis discusses his research into therapies that target the single strands of DNA or RNA which cause many cases of amyotrophic lateral sclerosis, also known as ALS or Lou Gehrig’s disease. His article “Phase 1–2 […]

VALOR: A Clinical Study for People Living with SOD1-ALS

Timothy M. Miller, MD, PhD, (Washington University) will discuss VALOR, a global, Phase 3 clinical study sponsored by Biogen for eligible people living with SOD1-ALS. VALOR is being conducted to evaluate the safety and potential efficacy of the investigational drug tofersen in SOD1-ALS. 

Promising Results for Investigational Treatments for Inherited ALS (Links to an external site)

Two preliminary studies were published simultaneously in the New England Journal of Medicine, both aimed at reducing superoxide dismutase 1 (SOD1) protein levels in people who had amyotrophic lateral sclerosis (ALS) due to SOD1 mutations. In the first study, a phase I/II trial of intrathecal tofersen, Timothy Miller, MD, PhD, of Washington University in St. […]

Update #2: Impact of COVID-19 on ALS Research

NEALS Educational Webinar. As the COVID-19 situation continues to evolve, we know it is of particular concern for the ALS community, both for clinical care and research trials. To help address some of your questions and concerns, representatives from the NEALS leadership team will be hosting a second webinar on the impact of COVID-19 on ALS clinical research.

Impact of COVID-19 on ALS Clinical Research

NEALS Educational Webinar. As the COVID-19 situation continues to evolve, we know it is of particular concern for the ALS community, both for clinical care and research trials.

Prospective natural history study of C9orf72 ALS clinical characteristics and biomarkers

Prospective natural history study of C9orf72ALS clinical characteristics and biomarkers Our team’s hard work has been published in Neurology. This paper profiles the clinical features, such as age at disease onset, survival duration, and measures of disease progression, of ALS patients with mutations in the C9orf72 gene.

Healey Center International Prize for Innovation in ALS Award (Links to an external site)

The award goes to Timothy Miller, MD, PhD, of Washington University School of Medicine in St. Louis, Don Cleveland, PhD, of the Ludwig Institute at the University of California at San Diego, Richard Smith, MD, of the Center of Neurological Study in La Jolla, California, Toby Ferguson, MD, PhD, for Biogen and Frank Bennett, PhD, […]

Gene-targeted Therapeutics for ALS (Links to an external site)

Presenter: Timothy Miller, MD, PhD. Novel gene-targeted therapies are being developed for ALS and other neurodegenerative diseases.  We will discuss how these drugs work and some of the research driving these clinical trials.

New Diagnostic Test for Neurofilament

Biomarkers are measures reflective of biological processes that occur in the body. In the setting of disease, biomarkers may be used for diagnostic, prognostic or treatment monitoring purposes.

Experimental Drug Shows Promise for Genetic Form of ALS (Links to an external site)

An early stage trial of an investigational therapy for amyotrophic lateral sclerosis (ALS) suggests that people could tolerate the experimental drug and, in exploratory results, the experimental drug was linked to possible slower progression in people with a genetic form of the disease caused by mutations in a gene called superoxide dismutase 1 (SOD1).

Dr. Timothy Miller Wins Sheila Essey Award for ALS Research (Links to an external site)

The ALS Association, in partnership with the AAN and the American Brain Foundation, are awarding research funding to Timothy M. Miller, M.D., Ph.D., the David Clayson Professor of Neurology from the Washington University School of Medicine in St. Louis. The award recognizes significant research contributions in the search for the causes, prevention, and cure for amyotrophic lateral sclerosis (ALS). Since 1996, The ALS Association and the American Academy of Neurology have jointly chosen recipients of the award.

Genetic Mutations Linked to Higher Proportion of ALS Cases Than Previously Believed (Links to an external site)

New research indicating genetic mutations may underlie more ALS cases than scientists originally thought. Dr. Miller’s close colleague Dr. Matthew Harms states, “To our surprise, we found that 26 percent of sporadic ALS patients had potential mutations in one of the known ALS genes we analyzed. This suggests that mutations may be contributing to significantly more ALS cases.”