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 […]
Emerging antisense oligonucleotide and viral therapies for amyotrophic lateral sclerosis (Links to an external site)
Ly CV, Miller TM.