Paula R. Clemens, MD

Professor of Neurology


  • Vice Chair, VA Affairs
  • Division Chief, Medical Service Line, VA – Pittsburgh Healthcare System

Biographical Sketch

Dr. Clemens’ clinical and research interests focus on the genetic myopathies. This translational research aims to identify and test treatments for genetic muscle conditions such as muscular dystrophy and late-onset Pompe disease. Dr. Clemens participates in and directs Phase II and Phase III clinical trials. Her current pre-clinical research is in collaboration with rheumatologist Dr. Dana Ascherman, focused on characterizing a mouse model of inflammatory myopathy.

Education & Training

  • Post-Doctorate, Baylor College of Medicine, Molecular Genetics
  • Post-Doctorate, Mayo Clinic – Rochester, Muscle Disease
  • Resident, Dartmouth Hitchcock Medical Center, Neurology
  • MD, The Medical College of Pennsylvania, Medicine

Specialized Areas of Clinical, Research and/or Educational Interests

  • Research: Genetic muscle disease
  • Clinical: Genetic muscle disease
  • Education: Research Training; Clinician-Educator and Investigator-Educator Career Development

Board Certifications

  • American Board of Psychiatry and Neurology

Professional Organization Membership

  • American Academy of Neurology
  • American Neurological Association
  • World Muscle Society
  • Medical Director, Cooperative International Neuromuscular Research Group (CINRG)

Honors & Awards

  • Curriculum Service Award, University of Pittsburgh School of Medicine, 2012
  • Clerkship Preceptor of the Year Award, University of Pittsburgh School of Medicine, 2011
  • Veterans Affairs VISN Annual Regional Award, Outstanding Researcher, 2005
  • Veterans Affairs Pittsburgh Healthcare System Basic Researcher of the Year Award, 2005

Selected Recent Publications

Hathout Y, Liang C, Ogundele M, Xu G, Tawalbeh SM, Dang UJ, Hoffman EP, Gordish-Dressman H, Conklin LS, van den Anker JN, Clemens PR, Mah JK, Henricson E, McDonald C. Disease-specific and glucocorticoid-responsive serum biomarkers for Duchenne Muscular Dystrophy. Sci Rep. 2019 Aug 21;9(1):12167. doi: 10.1038/s41598-019-48548-9. PMID: 31434957; PMCID: PMC6704115.

Mavroudis PD, van den Anker J, Conklin LS, Damsker JM, Hoffman EP, Nagaraju K, Clemens PR, Jusko WJ. Population Pharmacokinetics of Vamorolone (VBP15) in Healthy Men and Boys With Duchenne Muscular Dystrophy. J Clin Pharmacol. 2019 Jul;59(7):979-988. doi: 10.1002/jcph.1388. Epub 2019 Feb 11. PMID: 30742306; PMCID: PMC6548694.

Cudkowicz M, Chase MK, Coffey CS, Ecklund DJ, Thornell BJ, Lungu C, Mahoney K, Gutmann L, Shefner JM, Staley KJ, Bosch M, Foster E, Long JD, Bayman EO, Torner J, Yankey J, Peters R, Huff T, Conwit RA; NeuroNEXT Clinical Study Sites, Shinnar S, Patch D, Darras BT, Ellis A, Packer RJ, Marder KS, Chiriboga CA, Henchcliffe C, Moran JA, Nikolov B, Factor SA, Seeley C, Greenberg SM, Amato AA, DeGregorio S, Simuni T, Ward T, Kissel JT, Kolb SJ, Bartlett A, Quinn JF, Keith K, Levine SR, Gilles N, Coyle PK, Lamb J, Wolfe GI, Crumlish A, Mejico L, Iqbal MM, Bowen JD, Tongco C, Nabors LB, Bashir K, Benge M, McDonald CM, Henricson EK, Oskarsson B, Dobkin BH, Canamar C, Glauser TA, Woo D, Molloy A, Clark P, Vollmer TL, Stein AJ, Barohn RJ, Dimachkie MM, Le Pichon JB, Benatar MG, Steele J, Wechsler L, Clemens PR, Amity C, Holloway RG, Annis C, Goldberg MP, Andersen M, Iannaccone ST, Smith AG, Singleton JR, Doudova M, Haley EC, Quigg MS, Lowenhaupt S, Malow BA, Adkins K, Clifford DB, Teshome MA, Connolly N. Seven-Year Experience From the National Institute of Neurological Disorders and Stroke-Supported Network for Excellence in Neuroscience Clinical Trials. JAMA Neurol. 2020 Jun 1;77(6):755-763. doi: 10.1001/jamaneurol.2020.0367. PMID: 32202612; PMCID: PMC7483960.

Li X, Conklin LS, van den Anker J, Hoffman EP, Clemens PR, Jusko WJ. Exposure-Response Analysis of Vamorolone (VBP15) in Boys With Duchenne Muscular Dystrophy. J Clin Pharmacol. 2020 Oct;60(10):1385-1396. doi: 10.1002/jcph.1632. Epub 2020 May 20. PMID: 32434278; PMCID: PMC7494537.

Clemens PR, Rao VK, Connolly AM, Harper AD, Mah JK, Smith EC, McDonald CM, Zaidman CM, Morgenroth LP, Osaki H, Satou Y, Yamashita T, Hoffman EP; CINRG DNHS Investigators. Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial. JAMA Neurol. 2020 Aug 1;77(8):982-991. doi: 10.1001/jamaneurol.2020.1264. Erratum in: JAMA Neurol. 2020 Aug 1;77(8):1040. PMID: 32453377; PMCID: PMC7251505.