Boros BD, Schoch KM, Kreple CJ, Miller TM.
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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.
ALS Ice Bucket Challenge helps fund Washington University’s research
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.
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.”
ALS Trial Shows Novel Therapy is Safe (Links to an external site)
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.
Breakthrough ALS Research at Washington University (Links to an external site)
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.
Video: 2011 MDA Telethon ALS Research Update
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.
Video: 2010 MDA Telethon ALS Research Update
MDA presents an update regarding research on ALS, featuring Dr. Timothy M. Miller from Washington University in St. Louis.