Overview
Current Appointments & Affiliations
Assistant Research Professor Emeritus of Urology
·
1999 - Present
Surgery,
Clinical Science Departments
Recent Publications
Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial.
Journal Article JAMA Neurol · June 1, 2023 IMPORTANCE: In the previously reported Comparative Enzyme Replacement Trial With neoGAA Versus rhGAA (COMET) trial, avalglucosidase alfa treatment for 49 weeks showed clinically meaningful improvements in upright forced vital capacity (FVC) percent predict ... Full text Link to item CiteMotor Responses in Pediatric Pompe Disease in the ADVANCE Participant Cohort.
Journal Article J Neuromuscul Dis · 2022 BACKGROUND: ADVANCE (NCT01526785) presented an opportunity to obtain a more nuanced understanding of motor function changes in treatment-experienced children with Pompe disease receiving 4000L-production-scale alglucosidase alfa for 52 weeks. OBJECTIVE: To ... Full text Link to item CiteSafety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial.
Journal Article Lancet Neurol · December 2021 BACKGROUND: Pompe disease is a rare, progressive neuromuscular disorder caused by deficiency of acid α-glucosidase (GAA) and accumulation of lysosomal glycogen. We assessed the safety and efficacy of avalglucosidase alfa, a recombinant human GAA enzyme rep ... Full text Link to item CiteEducation, Training & Certifications
Iowa State University ·
1972
Ph.D.