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Amy D Harper, MD

Professor of Neurology
40 Ratings

Neurology, Neuromuscular clinic, Neuromuscular clinic

Board Certifications

Neuromuscular Medicine, Child Neurology


Children's Pavilion
1000 East Broad Street
Richmond, VA 23219
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Areas of interest

Pediatric neuromuscular disease
Muscular dystrophy
Charcot marie tooth
Neurodevelopmental disabilities
Neonatal neurodevelopment


Dr. Harper is an Associate Professor in the Department of Neurology, Division of Child Neurology. In 1998, she received her MD and entered pediatric residency. Following residency, Dr. Harper completed fellowships in neurodevelopmental disabilities/child neurology and neuromuscular medicine. She was the first neurodevelopmental disabilities training graduate nationwide. Her board certifications have included Pediatrics, Child Neurology and Neuromuscular Medicine.

In 2016, she joined the faculty of the Children’s Hospital of Richmond (CHOR)/VCU Health where she is the Shirley Van Epps Waple Professor. Clinically, her primary Her primary clinical leadership role is serving as Director of the CHOR Neuromuscular program which includes directorship of CHOR’s MDA, Duchenne Certified, and SMA Network Care Centers. Formerly, she was the founder, co-founder and/or director for multidisciplinary High Risk Neonatal, Cerebral Palsy, and Pediatric MDA clinics.

Dr. Harper’s contributions and expertise have been recognized on several levels. She was voted by her peers as “Top Doc” in Pediatric Neurology 6 times in 2 (2 states, cities (highest votes 2020). Neurology residents voted her “Best Teacher in Pediatric Neurology” in 2017. She has been recognized by the MDA, PPMD, Cure SMA, and the NC Epilepsy Foundation for her clinical care. Nationally, she serves as an Executive Committee member for the Collaborative Neuromuscular Research Group (CINRG). She has been an international workshop moderator, regional symposium course director, reviewer for 4 prominent journals, and contributor to book chapters.

Her research constitutes an unique portfolio. of neurodevelopment, neuro-genetics, and muscular dystrophy. Publications have focused on neurodevelopmental outcomes, natural history, novel genes, and new therapeutics in muscular dystrophy. She has led or actively collaborated on up to 21 ongoing research studies at a time. She has been co-investigator and Principal Investigator (PI) PI and Co-I on several industry, NIH, CDC and MDA sponsored muscular dystrophy, cerebral palsy, and high risk infant studies. She is involved in similar research efforts at VCU where she is PI and Co-I on up to 15 active studies. Muscular dystrophy research has included: translational collaborations, molecular diagnostics, PI for multi-center tissue banking (PI), and site PI CINRG associated clinical trials. Her research has contributed to CINRG related manuscripts, and supported the first FDA approved DMD gene modifying therapy for DMD. She is involved in similar research efforts at VCU, where she is PI and Co-I on up to 15 studies.

Dr. Harper’s clinical and research activities clearly demonstrate her dedicated passion for collaborative scholarship and clinical care in neuromuscular and neurodevelopmental disorders.

Major grant funding:

  • Astellas Pharma US, Inc. (0367-CL-0102) A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD), Role: PI
  • DSC/14/2357/51 Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study, Role: Limb Girdle 2I Ribitol study, MLBio, Role: PI
  • (DSC/14/2357/48) Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy, Italfarmaco, inVentiv Health Inc.
  • (Harper) (NS-065/NCNP-01-202) Open-Label, Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) (DMD), NS Pharma, Therapeutic Research in Neuromuscular Disorders Solutions, LLC (TRiNDS), Role: PI
  • Does Timing Matter? Efficacy of Parent Provided, Therapist Supported, Motor and Cognitive Intervention for Infants Born Very Preterm in the First Months of Life, National Institutes of Health, Role: Co-I 08/2018
  • Neuroimaging Feasibility for SPEEDI 2 Relationship Between CNS integrity and the Efficacy of a Parent Delivered Developmental Intervention for Infants Born Very Preterm. CCTR Endowment Fund Grant. Role: Co-I
  • (VBP15-004) A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) ReveraGen Biopharma, Inc, $231,203.00, Role: PI 04/2018 – Present
  • AVXS-101-RG-001, Registry, AveXis, Inc., Role: PI Network for Observational Study of Virginia Muscular Dystrophies (NoVA MD), Department of Health & Human Services, Role: Co-I Long-term Use of Viltolarsen in Boys with Duchenne Muscular Dystrophy in Clinical Practice. VILT-502, Preterm Brain Injury, neuroplasticity, and Response to Intervention: PremieNRTI, Role: Co-I


Undergraduate Education BA: University of Virginia
Medical School American University of the Caribbean
Residency Pediatrics: Vidant Medical Center Neurodevelopmental disabilities: Oregon Health and Science University Hospital
Fellowship Neuromuscular Fellowship, Oregon Health and Science University Hospital

Research interests

Muscular Dystrophies - DMD, LGMD, Myotonic Dystrophy, Congenital MD, Myopathy Spinal Muscular Atrophy


View publications here

  • Khurana, Sonia; Evans, Megan E; Kelly, Claire E; Thompson, Deanne; Burnsed, Jennifer; Harper, Amy; Munoz, Karen Hendricks; Shall, Mary S; Stevenson, Richard D.; Inamdar, Ketaki; Vorona, Gregory; Dusing, Stacey C Longitudinal changes in the sensorimotor pathways of very preterm infants during the first year of life with and without intervention: A pilot study. Developmental Neurorehabilitation, accepted
  • Sahajwalla D, Vorona G, Tye G, Harper A, Richard H, Sisler I, Ellett M, Cameron B, Rivet D, Urbine J. Aggressive vertebral hemangioma masquerading as neurological disease in a pediatric patient. Radiol Case Rep. 2021 May;16(5):1107-1112. doi: 10.1016/j.radcr.2021.02.023. eCollection 2021 May. PubMed PMID: 33732402; PubMed Central PMCID: PMC7937576.
  • Heier CR, Zhang A, Nguyen NY, Tully CB, Panigrahi A, Gordish-Dressman H, Pandey SN, Guglieri M, Ryan MM, Clemens PR, Thangarajh M, Webster R, Smith EC, Connolly AM, McDonald CM, Karachunski P, Tulinius M, Harper A, Mah JK, Fiorillo AA, Chen YW, Cooperative International Neuromuscular Research Group Cinrg Investigators. Multi-Omics Identifies Circulating miRNA and Protein Biomarkers for Facioscapulohumeral Dystrophy. J Pers Med. 2020 Nov 19;10(4). doi: 10.3390/jpm10040236. PubMed PMID: 33228131; PubMed Central PMCID: PMC7711540.
  • Clemens PR, Niizawa G, Feng J, Florence J, DʼAlessandro AS, Morgenroth LP, Gorni K, Guglieri M, Connolly A, Wicklund M, Bertorini T, Mah JK, Thangarajh M, Smith E, Kuntz N, McDonald CM, Henricson EK, Upadhyayula S, Byrne B, Manousakis G, Harper A, Bravver E, Iannaccone S, Spurney C, Cnaan A, Gordish-Dressman H. The CINRG Becker Natural History Study: Baseline characteristics. Muscle Nerve. 2020 Sep;62(3):369-376. doi: 10.1002/mus.27011. Epub 2020 Jul 24. PubMed PMID: 32564389; PubMed Central PMCID: PMC7520226.
  • Dusing SC, Burnsed JC, Brown SE, Harper AD, Hendricks-Munoz KD, Stevenson RD, Thacker LR, Molinini RM. Efficacy of Supporting Play Exploration and Early Development Intervention in the First Months of Life for Infants Born Very Preterm: 3-Arm Randomized Clinical Trial Protocol. Phys Ther. 2020 Aug 12;100(8):1343-1352. doi: 10.1093/ptj/pzaa077. PubMed PMID: 32329778; PubMed Central PMCID: PMC7439229.
  • 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. 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. PubMed PMID: 32453377; PubMed Central PMCID: PMC7251505
  • Donkervoort S, Sabouny R, Yun P, Gauquelin L, Chao KR, Hu Y, Al Khatib I, Töpf A, Mohassel P, Cummings BB, Kaur R, Saade D, Moore SA, Waddell LB, Farrar MA, Goodrich JK, Uapinyoying P, Chan SHS, Javed A, Leach ME, Karachunski P, Dalton J, Medne L, Harper A, Thompson C, Thiffault I, Specht S, Lamont RE, Saunders C, Racher H, Bernier FP, Mowat D, Witting N, Vissing J, Hanson R, Coffman KA, Hainlen M, Parboosingh JS, Carnevale A, Yoon G, Schnur RE, Boycott KM, Mah JK, Straub V, Foley AR, Innes AM, Bönnemann CG, Shutt TE. MSTO1 mutations cause mtDNA depletion, manifesting as muscular dystrophy with cerebellar involvement. Acta Neuropathol. 2019 Dec;138(6):1013-1031. doi: 10.1007/s00401-019- 02059-z. Epub 2019 Aug 29. PubMed PMID: 31463572; PubMed Central PMCID: PMC6851037.

Ratings & reviews

40 Ratings

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January 2021
good experience
July 2020
She explained everything in easy to understand terms, discussed follow up, and explained images as we reviewed them. It was extremely informative and helpful.
July 2020
She talked to me as well as my mother.
June 2020
Dr. Harper was/is great! Very thorough & kind. Timely & very informative in a caring way.
June 2020
Dr. Harper was fantastic.
  • Specialty: Pediatric Neurology Pediatric Neuromuscular Clinic Neurology Neuromuscula

Board Certifications

Clinical Interests

  • Pediatric neuromuscular disease
  • Muscular dystrophy
  • Charcot marie tooth
  • Neurodevelopmental disabilities
  • Neonatal neurodevelopment

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