Dr. Timothy Miller’s research focuses on bringing novel therapeutic strategies to neurodegenerative diseases. As we work toward the goal of bringing these new therapies to human clinical trial, our exciting research often attracts national and international attention.
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. […]
Tofersen demonstrates positive results in Phase I/II trial for amyotrophic lateral sclerosis
Biogen (MA, USA) has announced positive results from a Phase I/II study of tofersen – an antisense oligonucleotide – for the potential treatment of SOD1 amyotrophic lateral sclerosis (ALS). These results have been published in The New England Journal of Medicine.
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.
Research in St. Louis leads to promise in the fight against ALS
ST. LOUIS – Research taking place at Washington University’s School of Medicine is giving hope to those affected by amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.
Experimental drug shows early promise against inherited form of ALS, trial indicates
Phase 1/phase 2 trial shows evidence for safety, biological activity of tofersen; phase 3 trial underway
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).
Miller receives international innovation prize
Recognized for developing experimental treatment for 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, […]