A5043231646- Pharmaceutical Economics and Policy
- Health Systems, Economic Evaluations, Quality of Life
- Retinal Diseases and Treatments
- Genomics and Rare Diseases
- Ocular Oncology and Treatments
- Cancer Genomics and Diagnostics
- Pharmaceutical studies and practices
- Photodynamic Therapy Research Studies
- Ethics in Clinical Research
- Kidney Stones and Urolithiasis Treatments
- Leprosy Research and Treatment
- Peripheral Neuropathies and Disorders
- Cardiac electrophysiology and arrhythmias
- Cardiac pacing and defibrillation studies
- Botulinum Toxin and Related Neurological Disorders
- Cardiac Arrhythmias and Treatments
- CRISPR and Genetic Engineering
- Ocular Infections and Treatments
Amsterdam University Medical Centers
2022-2024
University of Amsterdam
2003-2024
Amsterdam Neuroscience
2024
Vrije Universiteit Amsterdam
2024
Since July 2021, a worldwide shortage of verteporfin (Visudyne®) occurred: an essential medicine required for photodynamic therapy (PDT). PDT with has broad range indications in ophthalmology, including chronic central serous chorioretinopathy, polypoidal choroidal vasculopathy and haemangioma. For these disorders, is either the first-choice treatment or regarded as major option.A questionnaire was sent to key opinion leaders field medical retina throughout world, assess role their country...
An ongoing global shortage of verteporfin (Visudyne
Progress in genetic diagnosis and orphan drug legislation has opened doors to new therapies rare neurogenetic diseases (RNDs). Innovative such as gene therapy can improve patients' quality of life but come with academic, regulatory, financial challenges. Registries play a pivotal role generating evidence tackle these, their development requires multidisciplinary knowledge expertise. This study aims develop practical framework for creating implementing patient registries addressing common...
Background: Novel or repurposed medicines for rare diseases often emerge from fundamental research empirical findings in academia. However, researchers may be insufficiently aware of the possibilities and requirements to bring novel medicinal treatment options patient. This paper aims provide an easily applicable, comprehensive roadmap designed academic make available patients by addressing relevant regulatory frameworks, including marketing authorization alternative routes. Methods: Key...
The objective of this study was to examine the clinical signs, symptoms and course neuropathies in patients with leprosy who after treatment developed nerve impairment, not explained by relapse or reversal reactions. We searched case-records patients, seen between 1985 2002 at department dermatology our centre. Included were had impairment absence relapse, erythema nodosum leprosum, reactions, referred a neurologist. In these we recorded age, onset leprosy, type signs delayed results...
Abstract Background The combination of high prices and uncertain effectiveness is a growing challenge in the field orphan medicines, hampering health technology assessments. Hence, new methods for establishing price benchmarks might be necessary to support reimbursement negotiations. In this study, we applied several pricing models containing cost-based elements case lumasiran treating primary hyperoxaluria type 1. Methods Price ranges were calculated by estimating minimum maximum scenarios...