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

Long-term follow-up of renal function in patients treated with migalastat for Fabry disease

by Daniel G. Bichet, Roser Torra, Eric Wallace, Derralynn Hughes, Roberto Giugliani, Nina Skuban, Eva Krusinska, Ulla Feldt-Rasmussen, Raphael Schiffmann, Kathy Nicholls
Published 2021-09-01

Wound closure in epidermolysis bullosa: data from the vehicle arm of the phase 3 ESSENCE Study

by Dedee F. Murrell, Amy S. Paller, Christine Bodemer, John Browning, Milos Nikolic, Jay A. Barth, Hjalmar Lagast, Eva Krusinska, Allen Reha, on behalf of the ESSENCE Study Group
Published 2020-07-01

Efficacy and tolerability of the investigational topical cream SD-101 (6% allantoin) in patients with epidermolysis bullosa: a phase 3, randomized, double-blind, vehicle-controlled...

by Amy S. Paller, John Browning, Milos Nikolic, Christine Bodemer, Dedee F. Murrell, Willistine Lenon, Eva Krusinska, Allen Reha, Hjalmar Lagast, Jay A. Barth, on behalf of the ESSENCE Study Group
Published 2020-06-01