At the American Neurological Association (ANA) Annual Meeting, Dr. Timothy Miller presented VALOR study of Tofersen Phase 3 Drug Results and Use with ALS. Tofersen is an antisense drug being evaluated for the potential treatment of SOD1-ALS. For more information and to learn about Tofersen Phase 3 Drug Results please see links below. Biogen News Release; […]
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)
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 […]
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. […]
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.
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.
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.
Phase 1/phase 2 trial shows evidence for safety, biological activity of tofersen; phase 3 trial underway
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.
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.
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).
Recognized for developing experimental treatment for ALS
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.
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, […]
With funds raised through the ALS Ice Bucket Challenge, The ALS Association has been able to invest significantly in the identification of biological indicators (or biomarkers) for ALS.
A genetic therapy that increases or lowers levels of a protein raises hopes for a treatment for neurological disorders.