Second generation registry framework
- 期刊名称Source Code for Biology and Medicine
- 作者Matthew I Bellgard,Lee Render,Maciej Radochonski…
- 卷第9卷
- 期第1期
- 电子ISSN1751-0473
- 文件大小437.56KB
- 发表时间2014-12
- DOI10.1186/1751-0473-9-14
ResponseAnnemieke Aartsma-Rus; Pat Furlong; Elizabeth Vroom; Gert-Jan van Ommen; Erik Niks; Chiara Straathof; Jan Verschuuren; Annemieke Aartsma-Rus; Alessandra Ferlini; Lynn Hagger; Emma Heslop; Veronika Karcagi; Jan Kirschner; Pauline McCormack; Pat Moeschen; Francesco Muntoni; Marie-Christine Ouillade; Jes Rahbeck; Christoph Rehmann-Sutter; Francoise Rouault; Tomas Sejersen; Elizabeth Vroom; Simon Woods · 2011
Letter 2Filippo Buccella; Pat Furlong; Sally Hofmeister; Elizabeth Vroom · 2010
2nd Workshop on upper-extremity assistive technology for people with Duchenne: Effectiveness and usability of arm supports Irvine, USA, 22nd–23rd January 2018Janssen, Mariska M.H.P.; Lobo-Prat, Joan; Bergsma, Arjen; Vroom, Elizabeth; Bergsma, Arjen; Reinkensmeyer, David; Vroom, Elizabeth; Han, Jay; Prat, Joan Lobo; Corrigan, Madeline; Janssen, Mariska; Paalman, Micha; Foulds, Richard; Bendixen, Roxanna; Rahman, Tariq · Neuromuscular Disorders · 2019
The risks of therapeutic misconception and individual patient (n = 1) “trials” in rare diseases such as Duchenne dystrophyAnnemieke Aartsma-Rus; Pat Furlong; Elizabeth Vroom; Gert-Jan van Ommen; Erik Niks; Chiara Straathof; Jan Verschuuren; Annemieke Aartsma-Rus; Alessandra Ferlini; Lynn Hagger; Emma Heslop; Veronika Karcagi; Jan Kirschner; Pauline McCormack; Pat Moeschen; Francesco Muntoni; Marie-Christine Ouillade; Jes Rahbeck; Christoph Rehmann-Sutter; Francoise Rouault; Tomas Sejersen; Elizabeth Vroom; Simon Woods · 2011
Biomarkers and surrogate endpoints in Duchenne: Meeting reportAartsma-Rus, Annemieke; Ferlini, Alessandra; Vroom, Elizabeth · Neuromuscular Disorders · 2014
Translational and Regulatory Challenges for Exon Skipping TherapiesAartsma-Rus, Annemieke; Ferlini, Alessandra; Goemans, Nathalie; Pasmooij, Anna M.G.; Wells, Dominic J.; Bushby, Katerine; Vroom, Elizabeth; Balabanov, Pavel · Human Gene Therapy · 2014
Looking under every rock: Duchenne muscular dystrophy and traditional Chinese medicineJ. Andoni Urtizberea; Qi-Shi Fan; Elizabeth Vroom; Dominique Récan; Jean-Claude Kaplan · 2003
Reviewer acknowledgement 2014Aymé, Ségolène · Orphanet Journal of Rare Diseases · 2014
National plans: case study BelgiumRi De Ridder · Orphanet Journal of Rare Diseases · 2012
Spina Bifida and primary preventionPierre Mertens · Orphanet Journal of Rare Diseases · 2012
Exon skipping for DMDAnnemieke Aartsma-Rus,Jan JGM Verschuuren… · Orphanet Journal of Rare Diseases · 2012
Gestational pemphigoidLaura Huilaja,Kaarin Mäkikallio,Kaisa Tasanen · Orphanet Journal of Rare Diseases · 2014
Ventricular septal defectSpicer, Diane E; Hsu, Hao H; Co-Vu, Jennifer; Anderson, Robert H; Fricker, F · Orphanet Journal of Rare Diseases · 2014
Managed entry agreementsMuscolo, Luisa AA; Bernardini, Claudia; Siviero, Paolo D; Montilla, Simona; Pani, Luca · Orphanet Journal of Rare Diseases · 2014
Empowerment Weekends for young adultsLemli, Annette; Schwarzer, Nicole · Orphanet Journal of Rare Diseases · 2014