Use of the patient-reported outcomes measurement information system (PROMIS®) to assess late-onset Pompe disease severity

by Melodi Harfouche, Priya S. Kishnani, Eva Krusinska, Jamie Gault, Sheela Sitaraman, Amanda Sowinski, Irina Katz, Stephanie Austin, Margi Goldstein, Andrew E. Mulberg
Published 2020-10-01

The challenges of living with and managing epidermolysis bullosa: insights from patients and caregivers

by Anna L. Bruckner, Michael Losow, Jayson Wisk, Nita Patel, Allen Reha, Hjalmar Lagast, Jamie Gault, Jayne Gershkowitz, Brett Kopelan, Michael Hund, Dedee F. Murrell
Published 2020-01-01