Timothy Miller, MD, PhD

Clayson Professor of Neurology

Board Certifications

  • Neurology - Certified
  • Psychiatry - Certified

Related Links

Clinical Interests

Dr. Timothy Miller performed his graduate studies with Dr. Eugene Johnson from 1993-1997 and completed his M.D./Ph.D. at Washington University School of Medicine in 1998. He then trained as a neurology resident and neuromuscular fellow at the University of California-San Francisco from 1998-2003. Afterwards, Dr. Miller moved to the University of California-San Diego where he spent four years as part of the Mentored Scientist Clinical Training Program doing post-doctoral work on disease models of ALS. Dr. Miller’s research focuses on methods to turn off harmful genes in the brain and spinal cord as a way to bring novel treatments to neurodegenerative diseases, in particular ALS. His other research interests include understand­ing how dysfunction of mitochondria, the power generators for cells, may be causing ALS. Dr. Miller has become a national leader in translational neuroscience and new therapeutic approaches for ALS.

As a board certified neurologist with a subspecialty in neurophysiology, Dr. Miller sees patients and participates in clinical trials in the Washington University Neuromuscular Clinic. He is currently an assistant professor of neurology at Washington University in St. Louis and a faculty member of the Hope Center for NeurologicalDisorders. He is the first director of the Christopher Wells Hobler Laboratory for ALS Research.


  • Cady J, Koval ED, Benitez BA, Zaidman C, Jockel-Balsarotti J, Allred P, Baloh RH, Ravits J, Simpson E, Appel SH, Pestronk A, Goate AM, Miller TM, Cruchaga C, Harms MB. TREM2 Variant p.R47H as a Risk Factor for Sporadic Amyotrophic Lateral Sclerosis. JAMA Neurol. 2014 Apr 1;71(4):449-53.
  • Crisp MJ, Beckett J, Coates JR, Miller TM. Canine degenerative myelopathy: biochemical characterization of superoxide dismutase 1 in the first naturally occurring non-human amyotrophic lateral sclerosis model. Exp Neurol. 2013 Oct;248:1-9.
  • Bali T, Miller TM. Management of amyotrophic lateral sclerosis. Mo Med. 2013 Sep-Oct;110(5):417-21. Review.
  • Holmes BB, DeVos SL, Kfoury N, Li M, Jacks R, Yanamandra K, Ouidja MO, Brodsky FM, Marasa J, Bagchi DP, Kotzbauer PT, Miller TM, Papy-Garcia D, Diamond MI. Heparan sulfate proteoglycans mediate internalization and propagation of specific proteopathic seeds. Proc Natl Acad Sci U S A. 2013 Aug 13;110(33):E3138-47.
  • DeVos SL, Goncharoff DK, Chen G, Kebodeaux CS, Yamada K, Stewart FR, Schuler DR, Maloney SE, Wozniak DF, Rigo F, Bennett CF, Cirrito JR, Holtzman DM, Miller TM. Antisense reduction of tau in adult mice protects against seizures. J Neurosci. 2013 Jul 31;33(31):12887-97.
  • DeVos SL, Miller TM. Antisense oligonucleotides: treating neurodegeneration at the level of RNA. Neurotherapeutics. 2013 Jul;10(3):486-97.
  • DeVos SL, Miller TM. Direct intraventricular delivery of drugs to the rodent central nervous system. J Vis Exp. 2013 May 12;(75):e50326.
  • Miller TM, Pestronk A, David W, Rothstein J, Simpson E, Appel SH, Andres PL, Mahoney K, Allred P, Alexander K, Ostrow LW, Schoenfeld D, Macklin EA, Norris DA, Manousakis G, Crisp M, Smith R, Bennett CF, Bishop KM, Cudkowicz ME. An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study. Lancet Neurol. 2013 May;12(5):435-42.
  • Winer L, Srinivasan D, Chun S, Lacomis D, Jaffa M, Fagan A, Holtzman DM, Wancewicz E, Bennett CF, Bowser R, Cudkowicz M, Miller TM. SOD1 in cerebral spinal fluid as a pharmacodynamic marker for antisense oligonucleotide therapy. JAMA Neurol. 2013 Feb;70(2):201-7.
  • Li Q, Vande Velde C, Israelson A, Xie J, Bailey AO, Dong MQ, Chun SJ, Roy T, Winer L, Yates JR, Capaldi RA, Cleveland DW, Miller TM. ALS-linked mutant superoxide dismutase 1 (SOD1) alters mitochondrial protein composition and decreases protein import. Proc Natl Acad Sci U S A. 2010 Dec 7;107(49):21146-51.
  • Harms MM, Miller TM, Baloh RH. TARDBP-Related Amyotrophic Lateral Sclerosis. In: Pagon RA, Adam MP, Ardinger HH, Bird TD, Dolan CR, Fong CT, Smith RJH, Stephens K, editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2014. 2009 Apr 23 [updated 2009 May 28].
  • Lopate G, Baloh RH, Al-Lozi MT, Miller TM, Fernandes Filho JA, Ni O, Leston A, Florence J, Schierbecker J, Allred P. Familial ALS with extreme phenotypic variability due to the I113T SOD1 mutation. Amyotroph Lateral Scler. 2010;11(1-2):232-6.
  • Wegorzewska I, Bell S, Cairns NJ, Miller TM, Baloh RH. TDP-43 mutant transgenic mice develop features of ALS and frontotemporal lobar degeneration. Proc Natl Acad Sci U S A. 2009 Nov 3;106(44):18809-14.
  • Miller TM. Differential diagnosis of myotonic disorders. Muscle Nerve. 2008 Mar;37(3):293-9. Review.
  • Miller TM, Moulder KL, Knudson CM, Creedon DJ, Deshmukh M, Korsmeyer SJ, Johnson EM Jr. Bax deletion further orders the cell death pathway in cerebellar granule cells and suggests a caspase-independent pathway to cell death. J Cell Biol. 1997 Oct 6;139(1):205-17.