Global Harmonization of Pediatric Drug Development: Critical for Progress for Developing Safe and Effective Therapeutic Agents for Children

by Andrew E. Mulberg, MD, FAAP, Timothy Cripps, MSc
Published 2019-01-01

Data silos are undermining drug development and failing rare disease patients

by Nathan Denton, Monique Molloy, Samantha Charleston, Craig Lipset, Jonathan Hirsch, Andrew E. Mulberg, Paul Howard, Eric D. Marsh
Published 2021-04-01

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

Regulatory strategies for rare diseases under current global regulatory statutes: a discussion with stakeholders

by Andrew E. Mulberg, Christina Bucci-Rechtweg, Joseph Giuliano, David Jacoby, Franklin K. Johnson, Qing Liu, Deborah Marsden, Scott McGoohan, Robert Nelson, Nita Patel, Klaus Romero, Vikram Sinha, Sheela Sitaraman, John Spaltro, Vivian Kessler
Published 2019-02-01