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"text": "Des signes de réaction allergique sévère, comme des difficultés respiratoires, sont urgents."
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"name": "La déferoxamine affecte-t-elle la vision ?",
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"name": "Comment prévenir la surcharge en fer ?",
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"text": "Éviter les transfusions sanguines inutiles et surveiller les niveaux de fer régulièrement."
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"name": "Les régimes alimentaires peuvent-ils influencer la surcharge en fer ?",
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"@type": "Question",
"name": "La déferoxamine peut-elle être utilisée préventivement ?",
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"text": "Elle est généralement utilisée pour traiter, mais peut être envisagée dans certains cas préventifs."
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"@type": "Question",
"name": "Comment la déferoxamine est-elle administrée ?",
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"name": "Quelle est la durée du traitement par déferoxamine ?",
"position": 17,
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"@type": "Question",
"name": "Peut-on combiner déferoxamine avec d'autres traitements ?",
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"@type": "Question",
"name": "Quels sont les objectifs du traitement par déferoxamine ?",
"position": 19,
"acceptedAnswer": {
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"text": "Réduire la surcharge en fer et prévenir les complications associées."
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{
"@type": "Question",
"name": "La déferoxamine est-elle efficace pour tous les patients ?",
"position": 20,
"acceptedAnswer": {
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"text": "Son efficacité peut varier selon la cause de la surcharge en fer et la réponse individuelle."
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{
"@type": "Question",
"name": "Quelles complications peuvent survenir avec une surcharge en fer ?",
"position": 21,
"acceptedAnswer": {
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"text": "Les complications incluent des maladies cardiaques, des troubles hépatiques et endocriniens."
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{
"@type": "Question",
"name": "La déferoxamine peut-elle causer des complications ?",
"position": 22,
"acceptedAnswer": {
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"text": "Oui, des complications comme des réactions allergiques et des troubles rénaux peuvent survenir."
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"@type": "Question",
"name": "Comment prévenir les complications liées à la surcharge en fer ?",
"position": 23,
"acceptedAnswer": {
"@type": "Answer",
"text": "Un traitement précoce et un suivi régulier des niveaux de fer sont essentiels."
}
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{
"@type": "Question",
"name": "Quels sont les risques à long terme d'une surcharge en fer ?",
"position": 24,
"acceptedAnswer": {
"@type": "Answer",
"text": "Les risques incluent des dommages organiques permanents, notamment au cœur et au foie."
}
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{
"@type": "Question",
"name": "La déferoxamine peut-elle réduire les complications ?",
"position": 25,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, elle aide à réduire la surcharge en fer et donc à diminuer les complications associées."
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Extremely high prices facilitate drug development for ultra-rare diseases (ultra-orphan drugs). However, various problems arise in terms of healthcare financing and fairness, and the status of ultra-o...
Drug prices tended to increase as the expected number of patients to whom the drug was administered decreased; however, this trend diminished when the expected number of patients was less than 1000. O...
The status of drug prices and expected sales differed beyond a threshold of 1000 expected patients, indicating that recovering the development cost for ultra-orphan drugs is difficult. In addition, th...
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More effective incentives are needed to motivate paediatric oncology drug development, uncoupling it from dependency on adult drug development. Although the current European and North-American legisla...
We propose changes in the timing and nature of the rewards within the European Paediatric Medicine Regulation (PMR) and Regulation on Orphan Medicinal Products (both currently under review), which wou...
We suggest modifying the PMR to ensure mechanism-of-action driven mandatory PIP and reorganization of incentives to a stepwise and incremental approach. Interim and final deliverables should be define...
Additionally, to address the critical gap in the current framework where there is a complete lack of incentives to promote paediatric-specific cancer drug development, we propose the introduction of e...
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While the drug development literature provides numerous estimates of the financial costs to bring a new drug to market, the investment of patient-participants in the research process has not been desc...
Osteoarthritis (OA) is the most common form of arthritis worldwide, affecting ~500 million people, yet there are no effective treatments to halt its progression. Without any structure-modifying agents...