Miller TM, Cudkowicz ME, Shaw PJ, Genge A, Sobue G, Bucelli RC, Chio A, Van Damme P, Ludolph AC, Glass JD, Andrews JA, Babu S, Benatar M, McDermott CJ, Salachas F, Bruneteau G, Al-Chalabi A, Amorin M, Nestorov I, Graham D, Lin L, Sun P, McNeill M, Malek S, Inra J, Garafalo S, Fradette S, […]
Author: admin
New ALS drug stabilizes decline with improved strength, mobility for some (Links to an external site)
Long-term use of tofersen slows ALS disease progression in people with a genetic subtype, provides hope for treating other forms of ALS. Read more »
Still standing: Inside a new era of ALS treatment (Links to an external site)
At dawn in rural Saskatchewan, the air is cool and damp, the grain still heavy with dew. Combines sit idle, waiting for the sun to dry out the field to make harvest possible. When it does, the machines will roar to life and work will stretch well past sunset.
New drug may slow progression of ALS in small group of patients: Study (Links to an external site)
SOD1-ALS from Gene Discovery to Targeted Therapeutics: A Comprehensive Review (Links to an external site)
Joshua Newman, Phillipe Corcia, Angela Genge, Robert Bucelli
When Getting Back to Normal is a Miracle (Links to an external site)
Our physician-scientists listen deeply, investigate thoroughly and bring answers to the unknown — never giving up until they’re able to deliver breakthroughs that improve our patients’ lives.
How did I train?
Getting ready for the ride, I started from a baseline of 2 swims, 1 weight lift, and 6-7 days a week on a bike for about 60-70 miles total for the week. To get ready for this ride, I tried to figure out how much mileage I would need to add to be ready for […]
2025 ALS Center Symposium
View session recordings.
Associations between end-stage ALS care and specialty palliative care: A hypothesis-generating study (Links to an external site)
Christi M. Lero, Annabelle Yang, Elyse Everett, Kyle A. Pitzer, Kelly McCoy Gross, Karla T. Washington
PODCAST: Revolutionizing ALS Treatment: The Tofersen Breakthrough (Links to an external site)
Dr. Gordon Smith talks with Dr. Timothy Miller about compelling patient stories and clinical data that highlight the potential for recovery in ALS patients, emphasizing the importance of early intervention and understanding the disease mechanisms.
Tofersen treatment leads to sustained stabilization of disease in SOD1 ALS in a “real-world” setting (Links to an external site)
Sean E. Smith, Kelly McCoy-Gross, Amber Malcolm, Jeri Oranski, Jesse W. Markway, Timothy M. Miller, Robert C. Bucelli
ALS Center Hosts Inaugural ALS Symposium
The ALS Center hosted its inaugural ALS Symposium on August 3, 2024, at the WashU Medical School campus.
Proceedings of the 23rd Annual NEALS Meeting (Links to an external site)
2024 ALS Center Symposium
View session recordings.
Open Fellowship Position in Neuromuscular Pathology at WashU School of Medicine (Links to an external site)
The WUSM Neuromuscular Pathology Fellowship is a non-ACGME fellowship focused on neuromuscular pathology and neuromuscular lab medicine. Learn more & application information
Lumbar punctures are safe in patients with ALS and have a similar risk profile relative to the non-ALS population (Links to an external site)
Kreple CJ, Gajagowni S, Jockel-Balsarotti J, Bucelli RC, Miller TM.
COURAGE-ALS: a randomized, double-blind phase 3 study designed to improve participant experience and increase the probability of success (Links to an external site)
Shefner JM, Al-Chalabi A, Andrews JA, Chio A, De Carvalho M, Cockroft BM, Corcia P, Couratier P, Cudkowicz ME, Genge A, Hardiman O, Heiman-Patterson T, Henderson RD, Ingre C, Jackson CE, Johnston W, Lechtzin N, Ludolph A, Maragakis NJ, Miller TM, Mora Pardina JS, Petri S, Simmons Z, Van Den Berg LH, Zinman L, Kupfer […]
Protective effects of lovastatin in a population-based ALS study and mouse model (Links to an external site)
Kreple CJ, Searles Nielsen S, Schoch KM, Shen T, Shabsovich M, Song Y, Racette BA, Miller TM.
Amyotrophic Lateral Sclerosis Clinical Trials and Interpretation of Functional End Points and Fluid Biomarkers: A Review (Links to an external site)
Shefner JM, Bedlack R, Andrews JA, Berry JD, Bowser R, Brown R, Glass JD, Maragakis NJ, Miller TM, Rothstein JD, Cudkowicz ME.
Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS (Links to an external site)
Miller TM, Cudkowicz ME, Genge A, Shaw PJ, Sobue G, Bucelli RC, Chiò A, Van Damme P, Ludolph AC, Glass JD, Andrews JA, Babu S, Benatar M, McDermott CJ, Cochrane T, Chary S, Chew S, Zhu H, Wu F, Nestorov I, Graham D, Sun P, McNeill M, Fanning L, Ferguson TA, Fradette S.
Antisense Oligonucleotides for the Study and Treatment of ALS (Links to an external site)
Boros BD, Schoch KM, Kreple CJ, Miller TM.
Prescription and acceptance of durable medical equipment in FORTITUDE-ALS, a study of reldesemtiv in ALS: post hoc analyses of a randomized, double-blind, placebo-controlled clinical trial (Links to an external site)
Rudnicki SA, Andrews JA, Genge A, Jackson C, Lechtzin N, Miller TM, Cockroft BM, Malik FI, Meng L, Wei J, Wolff AA, Shefner JM; FORTITUDE-ALS STUDY GROUP.
A Phase 2, Double-Blind, Randomized, Dose-Ranging Trial of Reldesemtiv in Patients with ALS (Links to an external site)
Shefner JM, Andrews JA, Genge A, Jackson C, Lechtzin N, Miller TM, et al.
Effect of sodium phenylbutyrate/taurursodiol on tracheostomy/ventilation-free survival and hospitalisation in amyotrophic lateral sclerosis: long-term results from the CENTAUR trial (Links to an external site)
Paganoni S, Hendrix S, Dickson SP, Knowlton N, Berry JD, Elliott MA, Maiser S, Karam C, Caress JB, Owegi MA, Quick A, Wymer J, Goutman SA, Heitzman D, Heiman-Patterson TD, Jackson C, Quinn C, Rothstein JD, Kasarskis EJ, Katz J, Jenkins L, Ladha SS, Miller TM, Scelsa SN, Vu TH, Fournier C, Johnson KM, Swenson […]
Using Smartphones to Reduce Research Burden in a Neurodegenerative Population and Assessing Participant Adherence: A Randomized Clinical Trial and Two Observational Studies (Links to an external site)
Beukenhorst AL, Burke KM, Scheier Z, Miller TM, Paganoni S, Keegan M, Collins E, Connaghan KP, Tay A, Chan J, Berry JD, Onnela JP.
Answer ALS, a large-scale resource for sporadic and familial ALS combining clinical and multi-omics data from induced pluripotent cell lines (Links to an external site)
Answer ALS is a biological and clinical resource of patient-derived, induced pluripotent stem (iPS) cell lines, multi-omic dataderived from iPS neurons and longitudinal clinical and smartphone data from over 1,000 patients with ALS. This resource provides population-level biological and clinical data that may be employed to identify clinical–molecular–biochemical subtypes ofamyotrophic lateral sclerosis (ALS). A unique […]
Is presymptomatic ALS perivascular? (Links to an external site)
Kreple, CJ, Schoch KM, Miller TM.
Smartphone data during the COVID-19 pandemic can quantify behavioral changes in people with ALS (Links to an external site)
Beukenhorst AL, Collins E, Burke KM, Rahman SM, Clapp M, Konanki SC, Paganoni S, Miller TM, Chan J, Onnela JP, Berry JD. Feb;63(2):258-262. PMID: 33118628.
Long-term survival of participants in the CENTAUR trial of sodium phenylbutyrate-taurursodiol in amyotrophic lateral sclerosis (Links to an external site)
Paganoni S, Hendrix S, Dickson SP, Knowlton N, Macklin EA, Berry JD, Elliott MA, Maiser S, Karam C, Caress JB, Owegi MA, Quick A, Wymer J, Goutman SA, Heitzman D, Heiman-Patterson TD, Jackson CE, Quinn C, Rothstein JD, Kasarskis EJ, Katz J, Jenkins L, Ladha S, Miller TM, Scelsa SN, Vu TH, Fournier CN, Glass […]
Trial of Sodium Phenylbutyrate-Taurursodiol for Amyotrophic Lateral Sclerosis (Links to an external site)
Paganoni S, Macklin EA, Hendrix S, Berry JD, Elliott MA, Maiser S, Karam C, Caress JB, Owegi MA, Quick A, Wymer J, Goutman SA, Heitzman D, Heiman-Patterson T, Jackson CE, Quinn C, Rothstein JD, Kasarskis EJ, Katz J, Jenkins L, Ladha S, Miller TM, Scelsa SN, Vu TH, Fournier CN, Glass JD, Johnson KM, Swenson […]
Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS (Links to an external site)
Miller T, Cudkowicz M, Shaw P, Andersen P, Nazem A, Bucelli R, Genge A, Glass J, Ladha S, Ludolph A, Maragakis N, McDermott C, Pestronk A, Ravits J, Salachas F, Trudell R, Van Damme P, Zinman L, Bennett CF, Lane R, Sandrock A, Runz H, Graham D, Houshyar H, McCampbell A, Nestorov I, Chang I, […]
Prospective natural history study of C9orf72 ALS clinical characteristics and biomarkers (Links to an external site)
Cammack AJ, Atassi N, Hyman T, van den Berg LH, Harms M, Baloh RH, Brown RH, van Es MA, Veldink JH, de Vries BS, Rothstein JD, Drain C, Jockel-Balsarotti J, Malcolm A, Boodram S, Salter A, Wightman N, Yu H, Sherman AV, Esparza TJ, McKenna-Yasek D, Owegi MA, Douthwright C, Alzheimer’s Disease Neuroimaging Initiative, McCampbell […]
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 (Links to an external site)
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).
Experimental treatment could be ‘game-changing’ for genetic ALS, experts say (Links to an external site)
(CNN) An experimental treatment for the rapidly progressive disease ALS, or amyotrophic lateral sclerosis, has been called potentially “game-changing.”
Video: C9 ID: Accelerating a therapy for ALS patients with C9 mutations
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.
Video: Advances in ALS Treatment
Washington University neurologist Timothy Miller, MD, discusses advances in ALS treatment.
Emerging antisense oligonucleotide and viral therapies for amyotrophic lateral sclerosis (Links to an external site)
Ly CV, Miller TM.
Dr. Timothy Miller Wins Sheila Essey Award for ALS Research (Links to an external site)
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.
Ste. Genevieve Riverdogs Baseball Team Supports ALS Research
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.
Medical Research Roundup: Dr. Tim Miller Awarded Research Grant from NIH (Links to an external site)
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.
Dr. Timothy Miller and Team Win the TDP-43 Biomarker Grand Challenge (Links to an external site)
The team is developing a unique imaging biomarker to track TDP-43, a protein found in almost all cases of ALS.
NEALS Webinar: “C9: From ID (Identification) to Therapy”
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.
Phase 1 Clinical Trial to Test BIIB067 in People with ALS Currently Recruiting Participants
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.
Genetic Mutations Linked to Higher Proportion of ALS Cases Than Previously Believed (Links to an external site)
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.”