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.
Biogen Announces Topline Results from the Tofersen Phase 3 Study and its Open-Label Extension in SOD1-ALS
View full story here: https://investors.biogen.com/news-releases/news-release-details/biogen-announces-topline-results-tofersen-phase-3-study-and-its View results from the Phase 3 VALOR study and its open-label extension at ANA 2021:https://biogen.gcs-web.com/static-files/b2154d4e-f69f-49d4-9a61-e834387293ea
Dr. Timothy Miller’s Featured Physicians Interview at Washington University
Check out Dr. Timothy Miller’s Featured Physicians interview, where WashU offers a more in-depth look at the people behind the white coats. Timothy Miller, MD, PhD – Washington University Physicians (wustl.edu)
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 […]
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. […]
Tofersen demonstrates positive results in Phase I/II trial for amyotrophic lateral sclerosis (Links to an external site)
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 (Links to an external site)
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 (Links to an external site)
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 (Links to an external site)
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.
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.
Simplifying the Search: How to Find the Right ALS Clinical Trial for You (Links to an external site)
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.
Healey ALS Platform Trial receives “May Proceed” notice for three drugs (Links to an external site)
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).