Orphan drug development: the increasing role of clinical pharmacology

Statistical modeling for Bayesian extrapolation of adult clinical trial information in pediatric drug evaluation

(2017) Margaret Gamalo-Siebers et al.   PHARMACEUTICAL STATISTICS

Alternative designs for clinical trials in rare diseases

(2016) Lusine Abrahamyan et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART C-SEMINARS IN MEDICAL GENETICS

A model-based approach to assess the exposure-response relationship of Lorenzo's oil in adrenoleukodystrophy

(2016) Mariam A. Ahmed et al.   BRITISH JOURNAL OF CLINICAL PHARMACOLOGY

Making Every Subject Count: A Case Study of Drug Development Path for Medication in a Pediatric Rare Disease

(2016) I Bhattacharya et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

FUTURE-2: Results from an open-label, long-term safety and tolerability extension study using the pediatric FormUlation of bosenTan in pUlmonary arterial hypeRtEnsion

(2016) Rolf M.F. Berger et al.   INTERNATIONAL JOURNAL OF CARDIOLOGY

A hierarchical Bayesian approach for combining pharmacokinetic/pharmacodynamic modeling and Phase IIa trial design in orphan drugs: Treating adrenoleukodystrophy with Lorenzo’s oil

(2016) Cynthia Basu et al.   Journal of Biopharmaceutical Statistics

2015 ESC/ERS Guidelines for the diagnosis and treatment of pulmonary hypertension

(2015) Nazzareno Galiè et al.   EUROPEAN HEART JOURNAL

Clinical trial designs for rare diseases: Studies developed and discussed by the International Rare Cancers Initiative

(2015) Jan Bogaerts et al.   EUROPEAN JOURNAL OF CANCER

STARTS-2

(2014) Robyn J. Barst et al.   CIRCULATION

A comparison of interventional clinical trials in rare versus non-rare diseases: an analysis of ClinicalTrials.gov

(2014) Stuart A Bell et al.   Orphanet Journal of Rare Diseases

A methodological framework for drug development in rare diseases

(2014) Patrice Nony et al.   Orphanet Journal of Rare Diseases

Clinical Trials in Rare Disease

(2013) Erika F. Augustine et al.   JOURNAL OF CHILD NEUROLOGY

Experimental designs for small randomised clinical trials: an algorithm for choice

(2013) Catherine Cornu et al.   Orphanet Journal of Rare Diseases

Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU

(2012) Roberta Joppi et al.   EUROPEAN JOURNAL OF CLINICAL PHARMACOLOGY

Ivacaftor potentiation of multiple CFTR channels with gating mutations

(2012) Haihui Yu et al.   Journal of Cystic Fibrosis

Biomarkers and surrogate endpoints in clinical trials

(2012) Thomas R. Fleming et al.   STATISTICS IN MEDICINE

A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study of Oral Sildenafil Citrate in Treatment-Naive Children With Pulmonary Arterial Hypertension

(2011) Robyn J. Barst et al.   CIRCULATION

Induction and Maintenance Therapy With Infliximab for Children With Moderate to Severe Ulcerative Colitis

(2011) Jeffrey Hyams et al.   Clinical Gastroenterology and Hepatology

Impact of Pharmacometric Analyses on New Drug Approval and Labelling Decisions

(2011) Joo Yeon Lee et al.   CLINICAL PHARMACOKINETICS

Characteristics of Clinical Trials to Support Approval of Orphan vs Nonorphan Drugs for Cancer

(2011) Aaron S. Kesselheim   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Outcome Following Infliximab Therapy in Children With Ulcerative Colitis

(2010) Jeffrey S Hyams et al.   AMERICAN JOURNAL OF GASTROENTEROLOGY

Applications of Physiologically Based Pharmacokinetic (PBPK) Modeling and Simulation During Regulatory Review

(2010) P Zhao et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Pivotal studies of orphan drugs approved for neurological diseases

(2009) Jun Mitsumoto et al.   ANNALS OF NEUROLOGY

Pharmacokinetic and clinical profile of a novel formulation of bosentan in children with pulmonary arterial hypertension: the FUTURE-1 study

(2009) Maurice Beghetti et al.   BRITISH JOURNAL OF CLINICAL PHARMACOLOGY

Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770

(2009) F. Van Goor et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Consensus statement on the management of pulmonary hypertension in clinical practice in the UK and Ireland

(2008)   HEART