About

Craig M. McDonald, M.D. is the Chair of the Department of Physical Medicine & Rehabilitation and a Professor in the Departments of Pediatrics and Physical Medicine & Rehabilitation at UC Davis Health. He specializes in neuromuscular medicine and pediatric rehabilitation medicine, with a focus on clinical management, rehabilitation, and precision therapeutics for children and adults with neuromuscular diseases. Dr. McDonald is an internationally recognized expert in the treatment and evaluation of children and young adults with Duchenne muscular dystrophy and other neuromuscular conditions. His research has concentrated on developing novel therapeutics for neuromuscular diseases, conducting clinical trials and natural history studies in muscular dystrophies, and utilizing outcome measures specifically designed for clinical trials. His work has significantly contributed to the development of precision-based therapeutics for Duchenne muscular dystrophy, including the first two therapies approved for targeting the gene abnormalities responsible for the disease. Dr. McDonald believes in team-based multidisciplinary care for patients with complex health challenges and aims to provide collaborative, coordinated care while pursuing innovative therapeutics to improve health, function, and quality of life.

Research topics

• Medicine
• Internal medicine
• Physical therapy
• Physical medicine and rehabilitation
• Pediatrics
• Biology
• Political Science
• Cardiology
• Surgery
• Radiology
• Cell biology
• Endocrinology

Selected publications

• Leading the Way

  American Journal of Physical Medicine & Rehabilitation · 2026-05-20

  article

  Regenerative rehabilitation combines rehabilitation science with regenerative medicine approaches to achieve synergistic and more effective outcomes. Within the field of regenerative medicine, there has been a growing number of cell and gene therapies (CGTs) that are either approved for clinical use or actively undergoing clinical trials. Many of these therapies-especially those targeting neurological and musculoskeletal disorders-use functional outcomes as primary or secondary endpoints, prompting discussion about how physical medicine and rehabilitation (PM&R) physicians can support the integration of CGTs into clinical care. In this perspective article, we explain why physicians specializing in PM&R could take a leading role in advancing the clinical translation of CGTs. Currently, CGTs are not confined to a single medical specialty, presenting a timely opportunity for PM&R-working collaboratively with neurology, hematology, and other disciplines-to assume greater leadership and influence the development and implementation of these transformative treatments. To our knowledge, this is the first publication within PM&R literature to emphasize the importance of our specialty serving as a leader and driving force in the adoption of these therapies.

  PublisherDOI

• Effectiveness of Ataluren in Patients with nmDMD: Confirmatory Evidence from the STRIDE Registry

  Neuropediatrics · 2025-09-26

  article
  PublisherDOI

• Givinostat in Duchenne Muscular Dystrophy: Natural history comparison applying propensity score matching

  Nervenheilkunde · 2025-03-01

  article
  PublisherDOI

• 680VPDisease progression modeling in Duchenne muscular dystrophy: delayed decay in 4-stair climb with givinostat compared with standard of care

  Neuromuscular Disorders · 2025-09-01

  article
  PublisherDOI

• Effectiveness of Ataluren in Patients with nmDMD: Confirmatory Evidence from the STRIDE Registry (P7-6.008)

  Neurology · 2025-04-07

  article

  To assess the reliability of ataluren effectiveness data in Strategic Targeting of Registries and International Database of Excellence (STRIDE [NCT02369731]) nonsense mutation DMD (nmDMD) patients and whether mutation type is a source of bias.

  PublisherDOI

• 424PDelandistrogene moxeparvovec micro-dystrophin expression and safety in 3–4-year-olds with Duchenne muscular dystrophy in ENDEAVOR and ENVOL studies

  Neuromuscular Disorders · 2025-09-01 · 1 citations

  article1st authorCorresponding
  PublisherDOI

• Long-Term Functional Outcomes, Safety, and Micro-Dystrophin Expression Following Delandistrogene Moxeparvovec Treatment in DMD: EMBARK 2-Year Results

  Neuropediatrics · 2025-09-26

  article
  PublisherDOI

• 181PAAV gene therapy with delandistrogene moxeparvovec in two-year old patients with Duchenne muscular dystrophy: clinical efficacy measured by digital endpoints

  Neuromuscular Disorders · 2025-09-01

  articleSenior author
  PublisherDOI

• Descriptive characterization of ambulatory health states in Duchenne muscular dystrophy: Motor function trajectories and times to loss of ambulation

  Journal of Neuromuscular Diseases · 2025-09-23

  articleOpen accessSenior author

  We described ambulatory Duchenne muscular dystrophy (DMD) progression, across multiple functional measures, via previously established prognostic groups for loss of ambulation (LoA) and health states. Patients closer to vs. farther from LoA had greater declines in some measures (e.g., 6-min walk distance) and less change in others (e.g., timed rise from floor velocity) due to floor effects. Patients in the late vs. early ambulatory health state were concordantly shifted towards higher LoA risk. Findings further characterize health states and prognostic factors in ambulatory DMD and highlight the importance of multiple measures of function to fully characterize disease progression.

  PublisherDOI

• Long‐Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History Comparisons

  Annals of Clinical and Translational Neurology · 2025-08-19 · 8 citations

  articleOpen access1st authorCorresponding

  OBJECTIVES: This ongoing, open-label extension study is evaluating the long-term safety, tolerability, and efficacy of givinostat, a Class I and II histone deacetylase inhibitor, in patients with Duchenne muscular dystrophy (DMD). METHODS: The recruited patients completed one of two prior clinical studies (one Phase 2 and one Phase 3 [EPIDYS]), receiving givinostat or placebo, or were successfully screened but not randomized into EPIDYS. All receive givinostat oral suspension open-label at a flexible, weight-based dose in addition to systemic corticosteroids, and attend visits every 4 months. RESULTS: A total of 194 patients are included in the current analyses, with a mean duration of givinostat exposure (excluding use in prior studies) of 559.6 days (SD 373.0); when including use in the prior studies, the maximum exposure to givinostat was > 8 years. Although the majority of patients reported ≥ 1 adverse event (169/194 [87.1%]), most were mild/moderate in severity, and the safety profile of givinostat was consistent with prior studies. Post hoc comparisons with natural history datasets (ImagingDMD and CINRG) suggest, in propensity matched populations, givinostat added to systemic corticosteroids significantly delayed the loss of the ability to rise from the floor, the loss of the ability to complete the 4-stair climb test, and the loss of ambulation (by medians of 2.0-3.3 years; all nominal p < 0.05). INTERPRETATION: Overall, the safety and tolerability of long-term administration of givinostat in patients with DMD was consistent with previous studies. Comparisons with natural history data suggest that givinostat delays the occurrence of major disease progression milestones. TRIAL REGISTRATION: EudraCT number: 2017-000397-10; ClinicalTrials.gov identifier: NCT03373968.

  PublisherOA PDFDOI

Frequent coauthors

• Nathalie Goemans

  KU Leuven

  572 shared

• Richard S. Finkel

  St. Jude Children's Research Hospital

  516 shared

• Alberto Dubrovsky

  Favaloro University

  459 shared

• Barry J. Byrne

  University of Florida

  448 shared

• Haluk Topaloğlu

  445 shared

• Krista Vandenborne

  University of Florida

  280 shared

• Richard J. Barohn

  239 shared

• John F. Brandsema

  236 shared

Education

• Master Rehabilitation Medicine, Rehanilitation Medicine

  University of Washington

  1992

• MD

  University of Washington

  1987

• AB, Human Biology

  Stanford University

  1982

Awards & honors

• UC Davis Hibbard Williams Extraordinary Achievement Award in…
• Selected for the 2023 Top 10 Clinical Research Achievement A…
• Selected as a “Castle Connolly Top Doctor” for 2023 by a Pee…
• Annual "Top Doctors in Sacramento" List by Sacramento Magazi…
• Carrell-Krusen Award, 42nd Annual Carrell-Krusen Neuromuscul…