Zobrazeno 1 - 10
of 47
pro vyhledávání: '"Elizabeth Vroom"'
Autor:
Annemieke Aartsma-Rus, Liesbeth De Waele, Saskia Houwen-Opstal, Janbernd Kirschner, Yvonne D. Krom, Eugenio Mercuri, Erik H. Niks, Volker Straub, Hermine A. van Duyvenvoorde, Elizabeth Vroom
Publikováno v:
Journal of Neuromuscular Diseases. 10:315-325
Antisense oligonucleotide (ASO) mediated exon skipping aims to reframe dystrophin transcripts for patients with Duchenne muscular dystrophy (DMD). Currently 4 ASOs have been approved by the Food and Drug Administration targeting exon 45, 51 and 53 ba
Autor:
Anna Sarkozy, Rosaline Quinlivan, John P. Bourke, Alessandra Ferlini, Inès Barthélémy, Linda H Cripe, Emily Reuben, Teresinha Evangelista, Anca Florian, Josh Gribnau, Lidia Gonzalez-Quereda, Michela Guglieri, Erik Niks, Rahul Phadke, Luisa Politano, Ros Quinlivan, John Vissing, Nicol Voermans, Elizabeth Vroom, Aleksandra Pietrusz, Fernanda Fortunato, Saskia Houwen
Publikováno v:
Neuromuscular Disorders, 33, 3, pp. 274-284
Neuromuscular Disorders, 33(3), 274-284. Elsevier Ltd.
Neuromuscular Disorders, 33, 274-284
Neuromuscular Disorders, 33(3), 274-284. Elsevier Ltd.
Neuromuscular Disorders, 33, 274-284
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Publikováno v:
The patient. 15(5)
Caregivers routinely inform medical and regulatory decision making in rare pediatric diseases. While differences in treatment preferences across caregivers and patients have been observed for Duchenne muscular dystrophy, this evidence was limited by
Publikováno v:
Nucleic Acid Therapeutics, 32(2), 118-122. MARY ANN LIEBERT, INC
The drug development process is a long and arduous one, especially for rare diseases. Patient and patient representatives can and should be involved in this process from an early stage, since they have the perspective of living with a disease on a da
Autor:
Mariska M.H.P. Janssen, Joan Lobo-Prat, Arjen Bergsma, Elizabeth Vroom, David Reinkensmeyer, Jay Han, Joan Lobo Prat, Madeline Corrigan, Mariska Janssen, Micha Paalman, Richard Foulds, Roxanna Bendixen, Tariq Rahman
Publikováno v:
Neuromuscular Disorders, 29, 651-656
Neuromuscular Disorders, 29, 8, pp. 651-656
Neuromuscular disorders, 29(8), 651-656. Elsevier
Neuromuscular Disorders, 29, 8, pp. 651-656
Neuromuscular disorders, 29(8), 651-656. Elsevier
Contains fulltext : 215829.pdf (Publisher’s version ) (Closed access)
Publikováno v:
Journal of Neuromuscular Diseases
Journal of Neuromuscular Diseases, 8, 1097-1108
Journal of Neuromuscular Diseases, 8(6), 1097-1108. IOS PRESS
Journal of Neuromuscular Diseases, 8, 6, pp. 1097-1108
Journal of Neuromuscular Diseases, 8, 1097-1108
Journal of Neuromuscular Diseases, 8(6), 1097-1108. IOS PRESS
Journal of Neuromuscular Diseases, 8, 6, pp. 1097-1108
Background: For patients with rare diseases such as Duchenne and Becker muscular dystrophy (DMD/BMD), access to their health data is key to being able to advocate for themselves and be in control of their care. Since 2018, the DMD/BMD patient communi
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::14c04b495bdb17bfe4fa0444ef3ff72f
https://doi.org/10.3233/jnd-210721
https://doi.org/10.3233/jnd-210721
Autor:
Pauline McCormack, Simon Woods, Annemieke Aartsma-Rus, Lynn Hagger, Agnes Herczegfalvi, Emma Heslop, Joseph Irwin, Janbernd Kirschner, Patrick Moeschen, Francesco Muntoni, Marie-Christine Ouillade, Jes Rahbek, Christoph Rehmann-Sutter, Francoise Rouault, Thomas Sejersen, Elizabeth Vroom, Volker Straub, Kate Bushby, Alessandra Ferlini
Publikováno v:
The Ethical Challenges of Emerging Medical Technologies ISBN: 9781003074984
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::038f272a86fc515722cee8d7c98cb0e1
https://doi.org/10.4324/9781003074984-23
https://doi.org/10.4324/9781003074984-23
Publikováno v:
Expert review of pharmacoeconomicsoutcomes research. 20(1)
Introduction: There is a groundswell of interest from patient, industry, and regulatory groups to rigorously and transparently integrate patient-voice into regulatory decision-making. Patient-focused drug development (PFDD) is an approach established
Autor:
Elizabeth Vroom, Ingrid E.C. Verhaart, Marta L. Fiorotto, Mirjam Franken-Verbeek, Lenie van den Engel-Hoek
Publikováno v:
Neuromuscular Disorders, 28, 8, pp. 680-689
Neuromuscular Disorders, 28, 680-689
Neuromuscular Disorders, 28, 680-689
Item does not contain fulltext
Publikováno v:
Neuromuscular Disorders. 28:619-623