Research Projects & Grants

  • Patient suffering from granulomatous amoebic encephalitis treated with miltefosine, Role: PI, Status: Completed

College/University

  • Intrathecal 2-Hydroxypropyl-ß-cyclodextrin for neurological decline in patients with Niemann-Pick disease type C1, Role: Investigator, Rush University Medical Center, (12/2024 - 12/2025) Status: Approved
  • Phase 2 Study of Levetiracetam for Neonatal Seizures, Role: Principal Investigator, University of California, San Diego, (09/2012 - 08/2015) Status: Closed

Federal

  • Pediatric TBI and DAI: Normal appearing brain is not normal, Role: Investigator, National Institutes of Health/DHHS, (06/2024 - 06/2025) Status: Approved
  • Environmental and genetic risk factors for pediatric multiple sclerosis, Role: Investigator, National Institute of Neurological Disorders and Stroke/NIH/DHHS, (12/2024 - 12/2025) Status: Approved
  • Therapeutic hypothermia after pediatric cardiac arrest (the THAPCA trials) version 1.12, Role: Investigator, National Heart, Lung, and Blood Institute/NIH/DHHS, (02/2017 - 02/2018) Status: Completed

For Profit Organization

  • An Open-label Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Inebilizumab in Pediatric Subjects with Neuromyelitis Optica Spectrum Disorder, Role: Investigator, Horizon Therapeutics, Inc., (04/2025 - 04/2026) Status: Approved
  • LONG-TERM FOLLOW-UP STUDY OF PATIENTS WITH SPINAL MUSCULAR ATROPHY RECEIVING RISDIPLAM TREATMENT, Role: PI, Genentech, Inc., (01/2024) Status: Approved
  • A phase III double-blind, randomized, placebo-controlled study assessing the efficacy, safety and tolerability of Idebenone in patients with Duchenne muscular dystrophy receiving glucocorticoid steroids (SNT-II-012), Role: PI, Santhera Pharmaceuticals (Switzerland) limited, (06/2020 - 06/2021) Status: Completed
  • A phase III open-label extension study to assess the long-term safety and efficacy of Idebenone in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study, Role: PI, Santhera Pharmaceuticals (Switzerland) limited, (06/2020 - 06/2021) Status: Completed
  • A phase 3, randomized, double-blind, placebo-controlled efficacy and safety study of Ataluren in patients with nonsense mutation Duchenne muscular dystrophy and open-label extension, Role: PI, PTC Therapeutics, Inc., (01/2020 - 01/2021) Status: Completed
  • A multi-center extension study to determine the long-term safety and efficacy of BG00012 in pediatric subjects with relapsing-remitting multiple sclerosis (109MS311), Role: Investigator, Biogen, Inc., (05/2018 - 05/2019) Status: Completed

Internal

  • Use of advanced cardiac therapies for patients with Duchenne Muscular Dystrophy, Role: Investigator, LLU Dept. of Pediatrics, (11/2017 - 11/2018) Status: Completed

Non-Profit Organization

  • Diet and relapses in pediatric Multiple Sclerosis, Role: Investigator, National Multiple Sclerosis Society, (11/2024 - 11/2025) Status: Approved
  • MDA Care Center at Loma Linda University, Role: PD/PI, Muscular Dystrophy Association, (07/2021 - 06/2027) Status: Awarded