Last Updated: 09/26/2026
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Found 39 publications
An Open Letter to the Food and Drug Administration and Pharmaceutical Industry Concerning Drug Approval for Children With Inflammatory Bowel Disease.
Journal: Inflammatory bowel diseases
Published: August 11, 2024
Correction to: Sharing is caring: a call for a new era of rare disease research and development.
Journal: Orphanet journal of rare diseases
Published: January 23, 2023
Coefficient of Fat Absorption to Measure the Efficacy of Pancreatic Enzyme Replacement Therapy in People With Cystic Fibrosis: Gold Standard or Coal Standard?
Journal: Pancreas
Published: June 13, 2022
Regulatory and Clinical Expert Perspective of the 2022 FDA Draft Guidance "Celiac Disease: Developing Drugs for Adjunctive Treatment to a Gluten-Free Diet".
Journal: Gastroenterology
Published: May 05, 2022
Sharing is caring: a call for a new era of rare disease research and development.
Journal: Orphanet journal of rare diseases
Published: February 16, 2022
FABry Disease Patient-Reported Outcome-GastroIntestinal (FABPRO-GI): A new Fabry disease-specific gastrointestinal outcomes instrument.
Journal: Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation
Published: April 12, 2021
Data silos are undermining drug development and failing rare disease patients.
Journal: Orphanet journal of rare diseases
Published: February 05, 2021
Use of the patient-reported outcomes measurement information system (PROMIS®) to assess late-onset Pompe disease severity.
Journal: Journal of patient-reported outcomes
Published: May 05, 2020
Extrapolation of Adult Efficacy to Pediatric Patients With Chemotherapy-Induced Nausea and Vomiting.
Extrapolation of Adult Efficacy to Pediatric Patients With Chemotherapy-Induced Nausea and Vomiting.
Journal: Journal of clinical pharmacology
Published: September 04, 2019
Global Harmonization of Pediatric Drug Development: Critical for Progress for Developing Safe and Effective Therapeutic Agents for Children.
Journal: Current therapeutic research, clinical and experimental
Published: January 16, 2019
Last Updated: 09/26/2026