Selected Grants
A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy (Start up)
Clinical TrialPrincipal Investigator · Awarded by Pfizer, Inc. · 2025 - 2040A Phase 3, Multinational, Long-Term Follow-Up Study to Evaluate Safety and Efficacy in Subjects Who Have Previously Received SRP-9001 in a Clinical study
Clinical TrialPrincipal Investigator · Awarded by Sarepta Therapeutics, Inc. · 2024 - 2031Unified Program for Therapeutics in Children
Inst. Training Prgm or CMEPreceptor · Awarded by National Institutes of Health · 2025 - 2030Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients with Duchenne Muscular Dystrophy
Clinical TrialPrincipal Investigator · Awarded by Catalyst Pharmaceuticals, Inc. · 2024 - 2029Alveolar injury and repair in Pompe Disease
ResearchPrincipal Investigator · Awarded by National Institutes of Health · 2025 - 2028Optimizing Gene Therapy for Respiratory Insufficiency in Duchenne Muscular Dystrophy
ResearchPrincipal Investigator · Awarded by National Heart, Lung, and Blood Institute · 2024 - 2028Stimulating Access to Research in Residency (StARR) - NHLBI
Inst. Training Prgm or CMECo-Principal Investigator · Awarded by National Heart, Lung, and Blood Institute · 2018 - 2028Medical Scientist Training Program
Inst. Training Prgm or CMEPreceptor · Awarded by National Institute of General Medical Sciences · 2022 - 2027Respiratory Characterization of DMD Mouse Models
ResearchPrincipal Investigator · Awarded by Sarepta Therapeutics, Inc. · 2019 - 2027Duke Program of Training in Pulmonary ReSearch to Promote, Engage and Retain Academic Researchers (PROSPER)
Inst. Training Prgm or CMEPreceptor · Awarded by National Heart, Lung, and Blood Institute · 2022 - 2026Stable therapy in Pompe disease through genome editing
ResearchCo Investigator · Awarded by National Institute of Arthritis and Musculoskeletal and Skin Diseases · 2021 - 2026Research Training in Allergy and Clinical Immunology
Inst. Training Prgm or CMEMentor · Awarded by University of North Carolina - Chapel Hill · 2000 - 2026Expanded Duchenne Muscular Dystrophy (DMD) Natural History Study: Longitudinal Study of the Relationship Between Impairment, Activity Limitation, Participation, and Qualify of Life
ResearchPrincipal Investigator · Awarded by University of California - Davis · 2025 - 2026Duke Center for Advancement of Child Health (CAtCH).
Inst. Training Prgm or CMETraining Director · Awarded by Eunice Kennedy Shriver National Institute of Child Health and Human Development · 2021 - 2026A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)
Clinical TrialPrincipal Investigator · Awarded by Sarepta Therapeutics, Inc. · 2023 - 2026A Non-Interventional Study Evaluating the Impact of TRIKAFTA® Initiation on Children and Caregivers of Children With Cystic Fibrosis Aged 2-5 Years in a Real-World Setting
Clinical TrialCo-Principal Investigator · Awarded by Vertex Pharmaceuticals Inc. · 2023 - 2025SMA Care Center Registry
ResearchPrincipal Investigator · Awarded by Cure SMA · 2019 - 2024Targeted Therapy for Pompe Disease
ResearchPrincipal Investigator · Awarded by National Institutes of Health · 2019 - 2024Novel Adjunctive Therapies for Pompe Disease
ResearchMentor · Awarded by National Institutes of Health · 2022 - 2023Epigenome Editing Technologies for Treating Diverse Disease
ResearchCo Investigator · Awarded by National Institutes of Health · 2019 - 2023Respiratory Dysfunction in an Optineurin knock out ALS mouse model
ResearchPrincipal Investigator · Awarded by National Institutes of Health · 2020 - 2022A Novel Vector to treat smooth muscle pathology in Pompe Disease
FellowshipPrincipal Investigator · Awarded by North Carolina Biotechnology Center · 2020 - 2022Award for a Pharmacist
ResearchPrincipal Investigator · Awarded by Cystic Fibrosis Foundation · 2019 - 2022Mental Health Screening in Duke Pediatric CF Patients
ResearchPrincipal Investigator · Awarded by Cystic Fibrosis Foundation · 2018 - 2021A Phase III, randomized, double-blind, placebo-controlled study of AeroVanc for the treatment of persistent methicillin-resistant Staphylococcus aureus lung infection in cystic fibrosis patients
Clinical TrialPrincipal Investigator · Awarded by Savara Inc. · 2017 - 2020A Phase I Study of the Safety of AAV2/8 LSPhGAA in Late-onset Pompe Disease
Clinical TrialCo Investigator · Awarded by National Institutes of Health · 2017 - 2020A Post-approval Observational Study to Evaluate the Long-term Effectiveness and Safety of Orkambi in US Patients who Completed Study VX12-809-105 Part A
ResearchPrincipal Investigator · Awarded by Vertex Pharmaceuticals Inc. · 2018 - 2019Cystic Fibrosis Foundation
ResearchPrincipal Investigator · Awarded by Cystic Fibrosis Foundation · 2018 - 2019Gene Therapy for Upper Airway and Respiratory Insufficiency in an ALS Mouse Model
ResearchPrincipal Investigator · Awarded by National Institutes of Health · 2016 - 2019Parker B. Francis Jo Rae Wright Award for Scientific Excellence
ResearchPrincipal Investigator · Awarded by Parker B. Francis Foundation · 2018 - 2019Novel Approaches for Correcting Respiratory Insufficiency in Pompe Disease
ResearchPrincipal Investigator · Awarded by National Institutes of Health · 2014 - 2019External Relationships
- National Institutes of Health (NIH)
This faculty member (or a member of their immediate family) has reported outside activities with the companies, institutions, or organizations listed above. This information is available to institutional leadership and, when appropriate, management plans are in place to address potential conflicts of interest.