Promising Results for Investigational Treatments for Inherited ALS

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. […]

Experimental drug shows promising results for rare form of ALS

Researchers led by a Washington University neurologist found promising results for an experimental drug to treat a rare, inherited form of amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s disease.

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.

Simplifying the Search: How to Find the Right ALS Clinical Trial for You

This webinar provides introductory-level instruction on how to search for ALS clinical trials. You will learn what resources to use to find open ALS clinical trials and where to look for specific information such as eligibility criteria, trial site locations, and if traveling is required.

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.

Healey ALS Platform Trial receives “May Proceed” notice for three drugs

The Sean M. Healey & AMG Center for ALS at Mass General has received approval from the U.S. Food and Drug Administration (FDA) to proceed with administering three proposed drug regimens in the HEALEY ALS Platform Trial – the first trial of its kind for amyotrophic lateral sclerosis (ALS).

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

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

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

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

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.

Medical Research Roundup: Dr. Tim Miller Awarded Research Grant from NIH

The Miller Lab has found differences between healthy people and people with amyotrophic lateral sclerosis (ALS) in biomolecules known as microRNAs. This project seeks to understand the microRNA differences and the effect of adjusting them to try to develop new diagnostic tests or treatments for ALS.

Genetic Mutations Linked to Higher Proportion of ALS Cases Than Previously Believed

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.”

ALS Trial Shows Novel Therapy is Safe

Highlights the exciting new results from Dr. Miller’s first Phase I trial of SOD1-targeting ASOs, stating that the investigational treatment for inherited forms of ALS has passed an early clinical trial for safety.

Breakthrough ALS Research at Washington University

Dr. Miller featured in news article and video about his Phase I clinical trial of antisense oligonucleotides targeting the SOD1 protein in ALS patients with SOD1 mutations.