Zobrazeno 1 - 10
of 10
pro vyhledávání: '"Sibren van den Berg"'
Autor:
Noa Rosenberg, Sibren van den Berg, Nina N. Stolwijk, Bart A. W. Jacobs, Hendrika C. Post, Anna M. G. Pasmooij, Saco J. de Visser, Carla E. M. Hollak
Publikováno v:
Frontiers in Pharmacology, Vol 14 (2023)
Background: Novel or repurposed medicines for rare diseases often emerge from fundamental research or empirical findings in academia. However, researchers may be insufficiently aware of the possibilities and requirements to bring novel medicinal trea
Externí odkaz:
https://doaj.org/article/b95f30a494ad44cca6e4f96a3d519b80
Autor:
Daphne H. Schoenmakers, Shanice Beerepoot, Sibren van den Berg, Laura Adang, Annette Bley, Jaap-Jan Boelens, Francesca Fumagalli, Wim G. Goettsch, Sabine Grønborg, Samuel Groeschel, Peter M. van Hasselt, Carla E. M. Hollak, Caroline Lindemans, Fanny Mochel, Peter G. M. Mol, Caroline Sevin, Ayelet Zerem, Ludger Schöls, Nicole I. Wolf
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 17, Iss 1, Pp 1-14 (2022)
Abstract Background Metachromatic Leukodystrophy (MLD) is a rare lysosomal disorder. Patients suffer from relentless neurological deterioration leading to premature death. Recently, new treatment modalities, including gene therapy and enzyme replacem
Externí odkaz:
https://doaj.org/article/8c393f5208e146328f0af2066de15352
Autor:
Carla E. M. Hollak, Sandra Sirrs, Sibren van den Berg, Vincent van der Wel, Mirjam Langeveld, Hanka Dekker, Robin Lachmann, Saco J. de Visser
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 15, Iss 1, Pp 1-7 (2020)
Abstract Independent disease registries for pre-and post-approval of novel treatments for rare diseases are increasingly important for healthcare professionals, patients, regulators and the pharmaceutical industry. Current registries for rare disease
Externí odkaz:
https://doaj.org/article/afbdd2fe69ca4f0281f87c332c225ba6
Publikováno v:
Frontiers in Pharmacology, Vol 12 (2021)
Background: The European Commission highlights in its Pharmaceutical Strategy the role of academic researchers in drug repurposing, especially in the development of orphan medicinal products (OMPs). This study summarizes the contribution of academia
Externí odkaz:
https://doaj.org/article/5d29ab2ef34e4d50816cea40710cffb9
Autor:
Frederick W. Thielen, Renaud J.S.D. Heine, Sibren van den Berg, Renske M. T. ten Ham, Carin A. Uyl-de Groot
Publikováno v:
Cytotherapy, 24(12), 1245-1258. Elsevier Inc.
Background aims: Drug prices are regarded as one of the most influential factors in determining accessibility and affordability to novel therapies. Cell and gene therapies such as OTL-200 (brand name: Libmeldy) and AVXS-101 (brand name: Zolgensma) wi
Autor:
Martijn H van der Ree, Laura van Dussen, Noa Rosenberg, Nina Stolwijk, Sibren van den Berg, Vincent van der Wel, Bart A W Jacobs, Arthur A M Wilde, Carla E M Hollak, Pieter G Postema
Publikováno v:
EP Europace. 24(11):1809-1823
Aims While mexiletine has been used for over 40 years for prevention of (recurrent) ventricular arrhythmias and for myotonia, patient access has recently been critically endangered. Here we aim to demonstrate the effectiveness and safety of mexiletin
Autor:
Noa Rosenberg, Nina N. Stolwijk, Sibren van den Berg, Joris J. Heus, Vincent van der Wel, Teun van Gelder, Annet M. Bosch, Saco J. de Visser, Carla E. M. Hollak
Publikováno v:
Journal of Inherited Metabolic Disease. WILEY
Journal of Inherited Metabolic Disease
Journal of Inherited Metabolic Disease
Medicine development for rare diseases, including inborn errors of metabolism (IEMs) is challenging. Many academic innovations fail to reach the patient, either by stranding in the translational stage or due to suboptimal patient access related to pr
Publikováno v:
International Journal of Technology Assessment in Health Care. 38:S89-S90
IntroductionBefore admission to the insured package, the price of a medicine is usually assessed on the basis of the value of the medicine for the patient: the effect size on health and survival must be in line with the costs. That seems like a fair
Autor:
Saco J. de Visser, Robin H. Lachmann, Vincent van der Wel, Mirjam Langeveld, Carla E. M. Hollak, Sandra Sirrs, Sibren van den Berg, Hanka Dekker
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 15, Iss 1, Pp 1-7 (2020)
Orphanet Journal of Rare Diseases
Orphanet journal of rare diseases, 15(1):235. BioMed Central
Orphanet Journal of Rare Diseases
Orphanet journal of rare diseases, 15(1):235. BioMed Central
Independent disease registries for pre-and post-approval of novel treatments for rare diseases are increasingly important for healthcare professionals, patients, regulators and the pharmaceutical industry. Current registries for rare diseases to eval
Autor:
Saco J. de Visser, Bas C. Stunnenberg, Vincent van der Wel, Sibren van den Berg, Carla E. M. Hollak, Pieter G. Postema, Martijn H. van der Ree, Lonneke Timmers
Publikováno v:
Value in health, 24(7), 925-929. Elsevier Limited
Value in Health, 24, 7, pp. 925-929
Value in Health, 24, 925-929
Value in Health, 24, 7, pp. 925-929
Value in Health, 24, 925-929
Objectives Mexiletine is a long-known drug used for the treatment of arrhythmias and repurposed in the 1980s for patients with nondystrophic myotonia (NDM). Recently, the price of mexiletine in Europe increased significantly after registration as an
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::81585791e81c45877867cde3930aeb5c
https://pure.amc.nl/en/publications/costbased-price-calculation-of-mexiletine-for-nondystrophic-myotonia(1108007c-ce8b-4e0e-adb9-162d3e0b33f6).html
https://pure.amc.nl/en/publications/costbased-price-calculation-of-mexiletine-for-nondystrophic-myotonia(1108007c-ce8b-4e0e-adb9-162d3e0b33f6).html