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 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. By defining the natural history of this patient […]
Recognized for developing experimental treatment for ALS
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.
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.
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).
(CNN) An experimental treatment for the rapidly progressive disease ALS, or amyotrophic lateral sclerosis, has been called potentially “game-changing.”
In this video Dr. Timothy Miller provides an excellent explanation of how ALS works in the body and what researchers are doing to bring a therapy to ALS patients, especially those with mutation in the C9ORF72 gene.
Washington University neurologist Timothy Miller, MD, discusses advances in ALS treatment.
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.
Prior to the final game of the season, the Ste. Genevieve Riverdogs baseball team surprised Coach Jeremy Hoog with a touching tribute as well as a donation to Dr. Timothy Miller’s ALS Research Lab in honor of Coach Hoog’s mother – and the Riverdogs’ biggest fan – Cheryl.
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.
The team is developing a unique imaging biomarker to track TDP-43, a protein found in almost all cases of ALS.
Dr. Timothy Miller is featured in this news release describing how the money raised in the ALS Ice Bucket Challenge has helped discover new genetic causes of this disease. KSDK TV St. Louis
Dr. Timothy Miller, Washington University, and Dr. Merit Cudkowicz, Massachusetts General Hospital, provide an update on the C9orf72 Natural History Study and discuss therapies in development for C9 patients.
MDA announces enrollment in the Phase I/II trial of SOD1 antisense oligonucleotides. Dr. Timothy Miller led the first trial of these SOD1 ASOs in ALS patients and is now the academic Principle Investigator for the Phase I/II trial, sponsored by Biogen Idec and Ionis Pharmaceuticals.
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.”
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.
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.
MDA presents an update regarding research on ALS, featuring Dr. Timothy M. Miller from Washington University in St. Louis. MDA is supporting a clinical trial of antisense therapy seeking to counteract the affects of a toxic version of the protein SOD1 in patients with an inherited form of ALS.
MDA presents an update regarding research on ALS, featuring Dr. Timothy M. Miller from Washington University in St. Louis.