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.