相关参考文献
注意：仅列出部分参考文献，下载原文获取全部文献信息。Series: Pragmatic trials and real world evidence: Paper 4. Informed consent
Shona Kalkman et al.
JOURNAL OF CLINICAL EPIDEMIOLOGY (2017)
Ten years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registry
Joseph Muenzer et al.
ORPHANET JOURNAL OF RARE DISEASES (2017)
Registries in European post-marketing surveillance: a retrospective analysis of centrally approved products, 2005-2013
Jacoline C. Bouvy et al.
PHARMACOEPIDEMIOLOGY AND DRUG SAFETY (2017)
The DIAN-TU Next Generation Alzheimer's prevention trial: Adaptive design and disease progression model
Randall J. Bateman et al.
ALZHEIMERS & DEMENTIA (2017)
Registry-based randomized controlled trials- what are the advantages, challenges, and areas for future research?
Guowei Li et al.
JOURNAL OF CLINICAL EPIDEMIOLOGY (2016)
Directions for new developments on statistical design and analysis of small population group trials
Ralf-Dieter Hilgers et al.
ORPHANET JOURNAL OF RARE DISEASES (2016)
The history and accomplishments of the ICGG Gaucher registry INTRODUCTION
Neal J. Weinreb et al.
AMERICAN JOURNAL OF HEMATOLOGY (2015)
Update in Cystic Fibrosis 2014
Thida Ong et al.
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE (2015)
Detection and investigation of temporal clusters of congenital anomaly in Europe: seven years of experience of the EUROCAT surveillance system
Helen Dolk et al.
EUROPEAN JOURNAL OF EPIDEMIOLOGY (2015)
Rare Disease Registries Classification and Characterization: A Data Mining Approach
Michele Santoro et al.
PUBLIC HEALTH GENOMICS (2015)
Factors associated with FEV1 decline in cystic fibrosis: analysis of the ECFS Patient Registry
Eitan Kerem et al.
EUROPEAN RESPIRATORY JOURNAL (2014)
A multicenter, open-label study evaluating safety and clinical outcomes in children (1.4-7.5 years) with Hunter syndrome receiving idursulfase enzyme replacement therapy
Roberto Giugliani et al.
GENETICS IN MEDICINE (2014)
Developing Core Outcome Measurement Sets for Clinical Trials: OMERACT Filter 2.0
Maarten Boers et al.
JOURNAL OF CLINICAL EPIDEMIOLOGY (2014)
The European Cystic Fibrosis Society Patient Registry: valuable lessons learned on how to sustain a disease registry
Laura Viviani et al.
ORPHANET JOURNAL OF RARE DISEASES (2014)
Sample size calculations in pediatric clinical trials conducted in an ICU: a systematic review
Stavros Nikolakopoulos et al.
TRIALS (2014)
The EPIRARE proposal of a set of indicators and common data elements for the European platform for rare disease registration
Domenica Taruscio et al.
ARCHIVES OF PUBLIC HEALTH (2014)
Development of the Performance of the Upper Limb module for Duchenne muscular dystrophy
Anna Mayhew et al.
DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY (2013)
Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU
Roberta Joppi et al.
EUROPEAN JOURNAL OF CLINICAL PHARMACOLOGY (2013)
The Randomized Registry Trial - The Next Disruptive Technology in Clinical Research?
Michael S. Lauer et al.
NEW ENGLAND JOURNAL OF MEDICINE (2013)
A pilot 'cohort multiple randomised controlled trial' of treatment by a homeopath for women with menopausal hot flushes
C. Relton et al.
CONTEMPORARY CLINICAL TRIALS (2012)
Potential endpoints for clinical trials in premanifest and early Huntington's disease in the TRACK-HD study: analysis of 24 month observational data
Sarah J. Tabrizi et al.
LANCET NEUROLOGY (2012)
The case for a global rare-diseases registry
Christopher B. Forrest et al.
LANCET (2011)
Pompe disease: Design, methodology, and early findings from the Pompe Registry
Barry J. Byrne et al.
MOLECULAR GENETICS AND METABOLISM (2011)
Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders
Carla E. M. Hollak et al.
ORPHANET JOURNAL OF RARE DISEASES (2011)
CONSORT 2010 Explanation and Elaboration: updated guidelines for reporting parallel group randomised trials
David Moher et al.
JOURNAL OF CLINICAL EPIDEMIOLOGY (2010)
A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease.
Ans T. van der Ploeg et al.
NEW ENGLAND JOURNAL OF MEDICINE (2010)
Pitfalls in the design and analysis of paediatric clinical trials: a case of a 'failed' multi-centre study, and potential solutions
Johanna H. van der Lee et al.
ACTA PAEDIATRICA (2009)
Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease
Marc Nicolino et al.
GENETICS IN MEDICINE (2009)
Recombinant human acid α-glucosidase -: Major clinical benefits in infantile-onset Pompe disease
P. S. Kishnani et al.
NEUROLOGY (2007)
A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease
Priya S. Kishnani et al.
JOURNAL OF PEDIATRICS (2006)
Advances toward an understanding of brainstem gliomas
SS Donaldson et al.
JOURNAL OF CLINICAL ONCOLOGY (2006)
Psoriasis therapy in real life: The need for registries
Marcus Schmitt-Egenolf
DERMATOLOGY (2006)
Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: A report from the Gaucher Registry
NJ Weinreb et al.
AMERICAN JOURNAL OF MEDICINE (2002)
分享论文
