Selected Grants
Understanding cognitive and neurological pathologies in infantile Pompe disease CNS
Clinical TrialPhysical Therapist · Awarded by Genzyme Corporation · 2022 - 2024Developing a management approach for patients with the "late-onset" Pompe disease GAA variant identified by newborn screening
ResearchInvestigator · Awarded by Amicus Therapeutics Corporation · 2018 - 2022Understanding cognitive and neurological pathologies in infantile Pompe disease CNS
Clinical TrialPhysical Therapist · Awarded by Genzyme Corporation · 2019 - 2021Electrical Impedance Myography and Muscle Ultrasound: Longitudinal Measures in Pompe Disease
Clinical TrialInvestigator · Awarded by Genzyme Corporation · 2016 - 2021GSD III Clinical Data Manuscript Preparation
ResearchInvestigator · Awarded by Ultragenyx Pharmaceutical · 2019 - 2021A 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 - 2020Respiratory Muscle Training in Individuals With Pompe Disease
ResearchInvestigator · Awarded by National Institutes of Health · 2016 - 2019Understanding cognitive and neurological pathologies in infantile Pompe disease
Clinical TrialPhysical Therapist · Awarded by Genzyme Corporation · 2016 - 2018Role of Whole Body MRI as a Non-Invasive Technique for Characterizing Disease and Treatment Efficacy in Infantile Onset Pompe disease
Clinical TrialPhysical Therapist · Awarded by Genzyme Corporation · 2016 - 2017Supplemental Funding for Phase 1/2 study of Clenbuterol for the Treatment of Pompe Disease
Clinical TrialCo Investigator · Awarded by Roivant Sciences, Inc. · 2015 - 2016Phase 1/2 Study of Clenbuterol for the Treatment of Pompe Disease
Clinical TrialCo Investigator · Awarded by Food and Drug Administration · 2013 - 2016Clinical Trial Planning in Pompe Disease
ResearchCo Investigator · Awarded by National Institutes of Health · 2013 - 2014External Relationships
- Amicus Therapeutics
- AskBio (Actus)
- Avexis, Inc
- Avexis, Inc
- Biogen Idec Inc.
- Biohaven Pharmaceuticals
- CINRG (Cooperative International Neuromuscular Research Group)
- CryoCell International
- Genzyme Corporation (Sanofi Aventis)
- IONIS Pharmaceuticals
- Marcus Foundation
- NS Pharma - (parent company is Nippon Shinyaku)
- PTC Therapeutics
- Pfizer Inc.
- ReveraGen
- Robertson Foundation
- Sarepta Therapeutics
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.