Detalles de la búsqueda
1.
FABry Disease Patient-Reported Outcome-GastroIntestinal (FABPRO-GI): A new Fabry disease-specific gastrointestinal outcomes instrument.
Qual Life Res
; 30(10): 2983-2994, 2021 Oct.
Artículo
en Inglés
| MEDLINE | ID: mdl-33914257
3.
Regulatory and Clinical Expert Perspective of the 2022 FDA Draft Guidance "Celiac Disease: Developing Drugs for Adjunctive Treatment to a Gluten-Free Diet".
Gastroenterology
; 164(3): 317-319, 2023 03.
Artículo
en Inglés
| MEDLINE | ID: mdl-35810780
4.
Global Harmonization of Pediatric Drug Development: Critical for Progress for Developing Safe and Effective Therapeutic Agents for Children.
Curr Ther Res Clin Exp
; 90: 109-112, 2019.
Artículo
en Inglés
| MEDLINE | ID: mdl-31388364
5.
Pediatric Extrapolation of Adult Efficacy to Children Is Critical for Efficient and Successful Drug Development.
Gastroenterology
; 163(1): 77-83, 2022 07.
Artículo
en Inglés
| MEDLINE | ID: mdl-35288113
6.
Pediatric Crohn Disease Clinical Outcome Assessments and Biomarkers: Current State and Path Forward for Global Collaboration.
J Pediatr Gastroenterol Nutr
; 64(3): 368-372, 2017 03.
Artículo
en Inglés
| MEDLINE | ID: mdl-27253661
7.
Neurocognitive clinical outcome assessments for inborn errors of metabolism and other rare conditions.
Mol Genet Metab
; 118(2): 65-9, 2016 06.
Artículo
en Inglés
| MEDLINE | ID: mdl-27132782
8.
Nutritional interventions in primary mitochondrial disorders: Developing an evidence base.
Mol Genet Metab
; 119(3): 187-206, 2016 11.
Artículo
en Inglés
| MEDLINE | ID: mdl-27665271
9.
Well-defined and reliable clinical outcome assessments for pediatric Crohn disease: a critical need for drug development.
J Pediatr Gastroenterol Nutr
; 60(6): 729-36, 2015 Jun.
Artículo
en Inglés
| MEDLINE | ID: mdl-25793905
10.
Phenylketonuria Scientific Review Conference: state of the science and future research needs.
Mol Genet Metab
; 112(2): 87-122, 2014 Jun.
Artículo
en Inglés
| MEDLINE | ID: mdl-24667081
11.
Alternate endpoints and clinical outcome assessments in pediatric ulcerative colitis registration trials.
J Pediatr Gastroenterol Nutr
; 58(1): 12-7, 2014 Jan.
Artículo
en Inglés
| MEDLINE | ID: mdl-24121142
12.
Steps toward harmonization for clinical development of medicines in pediatric ulcerative colitis-a global scientific discussion, part 1: efficacy endpoints and disease outcome assessments.
J Pediatr Gastroenterol Nutr
; 58(6): 679-83, 2014 Jun.
Artículo
en Inglés
| MEDLINE | ID: mdl-24866781
13.
Steps toward harmonization for clinical development of medicines in pediatric ulcerative colitis-a global scientific discussion, part 2: data extrapolation, trial design, and pharmacokinetics.
J Pediatr Gastroenterol Nutr
; 58(6): 684-8, 2014 Jun.
Artículo
en Inglés
| MEDLINE | ID: mdl-24866782
14.
Off-label use of medicine in pediatrics: focus on gastrointestinal diseases.
Curr Opin Pediatr
; 25(5): 612-7, 2013 Oct.
Artículo
en Inglés
| MEDLINE | ID: mdl-23995427
15.
Cross-sector sponsorship of research in eosinophilic esophagitis: a collaborative model for rational drug development in rare diseases.
J Allergy Clin Immunol
; 130(3): 613-6, 2012 Sep.
Artículo
en Inglés
| MEDLINE | ID: mdl-22857796
16.
Development of Celiac Disease Therapeutics: Report of the Third Gastroenterology Regulatory Endpoints and Advancement of Therapeutics Workshop.
Gastroenterology
; 151(3): 407-11, 2016 09.
Artículo
en Inglés
| MEDLINE | ID: mdl-27456385
17.
Coefficient of Fat Absorption to Measure the Efficacy of Pancreatic Enzyme Replacement Therapy in People With Cystic Fibrosis: Gold Standard or Coal Standard?
Pancreas
; 51(4): 310-318, 2022 04 01.
Artículo
en Inglés
| MEDLINE | ID: mdl-35695742
18.
Sharing is caring: a call for a new era of rare disease research and development.
Orphanet J Rare Dis
; 17(1): 389, 2022 10 27.
Artículo
en Inglés
| MEDLINE | ID: mdl-36303170
19.
Pharmacokinetics and tolerability of rabeprazole in children 1 to 11 years old with gastroesophageal reflux disease.
J Pediatr Gastroenterol Nutr
; 52(6): 691-701, 2011 Jun.
Artículo
en Inglés
| MEDLINE | ID: mdl-21478756
20.
Treatment of infants and toddlers with cystic fibrosis-related pancreatic insufficiency and fat malabsorption with pancrelipase MT.
J Pediatr Gastroenterol Nutr
; 53(1): 61-4, 2011 Jul.
Artículo
en Inglés
| MEDLINE | ID: mdl-21694537