Quels sont les principaux facteurs de risque du MPP+ ?
L'exposition à des pesticides et à des produits chimiques industriels augmente le risque.
Facteurs de risquePesticides
#2
L'âge est-il un facteur de risque pour le MPP+ ?
Oui, le risque d'effets néfastes du MPP+ augmente avec l'âge.
ÂgeRisque
#3
Les antécédents familiaux influencent-ils le risque ?
Oui, des antécédents familiaux de maladies neurodégénératives augmentent le risque.
Antécédents familiauxMaladies neurodégénératives
#4
Le sexe joue-t-il un rôle dans le risque de MPP+ ?
Oui, des études montrent que les hommes sont plus à risque que les femmes.
SexeRisque
#5
Le mode de vie influence-t-il le risque de MPP+ ?
Oui, un mode de vie sédentaire et une mauvaise alimentation peuvent augmenter le risque.
Mode de vieAlimentation
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"name": "Comment diagnostiquer l'exposition au MPP+ ?",
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"acceptedAnswer": {
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"text": "Des tests biologiques et des évaluations neurologiques sont utilisés pour diagnostiquer l'exposition."
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{
"@type": "Question",
"name": "Quels examens sont recommandés pour le MPP+ ?",
"position": 2,
"acceptedAnswer": {
"@type": "Answer",
"text": "L'IRM et l'électromyographie peuvent aider à évaluer les dommages neurologiques."
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"@type": "Question",
"name": "Quels symptômes indiquent une intoxication au MPP+ ?",
"position": 3,
"acceptedAnswer": {
"@type": "Answer",
"text": "Des tremblements, rigidité et bradykinésie peuvent indiquer une intoxication au MPP+."
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"@type": "Question",
"name": "Le MPP+ peut-il être détecté dans le sang ?",
"position": 4,
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"text": "Oui, des analyses sanguines peuvent détecter la présence de MPP+."
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"@type": "Question",
"name": "Quels tests neurologiques sont utiles pour le MPP+ ?",
"position": 5,
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"@type": "Question",
"name": "Quels sont les symptômes principaux du MPP+ ?",
"position": 6,
"acceptedAnswer": {
"@type": "Answer",
"text": "Les symptômes incluent tremblements, rigidité musculaire et troubles de la marche."
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"@type": "Question",
"name": "Le MPP+ cause-t-il des troubles cognitifs ?",
"position": 7,
"acceptedAnswer": {
"@type": "Answer",
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"@type": "Question",
"name": "Y a-t-il des symptômes non moteurs liés au MPP+ ?",
"position": 8,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, des symptômes comme la dépression et l'anxiété peuvent survenir."
}
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"@type": "Question",
"name": "Comment le MPP+ affecte-t-il le système nerveux ?",
"position": 9,
"acceptedAnswer": {
"@type": "Answer",
"text": "Le MPP+ endommage les neurones dopaminergiques, entraînant des symptômes parkinsoniens."
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{
"@type": "Question",
"name": "Le MPP+ provoque-t-il des douleurs ?",
"position": 10,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, des douleurs musculaires et articulaires peuvent être présentes chez les patients."
}
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"@type": "Question",
"name": "Comment prévenir l'exposition au MPP+ ?",
"position": 11,
"acceptedAnswer": {
"@type": "Answer",
"text": "Éviter les environnements contaminés et utiliser des équipements de protection."
}
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{
"@type": "Question",
"name": "Y a-t-il des vaccins contre le MPP+ ?",
"position": 12,
"acceptedAnswer": {
"@type": "Answer",
"text": "Actuellement, il n'existe pas de vaccin spécifique contre le MPP+."
}
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{
"@type": "Question",
"name": "Les changements de mode de vie aident-ils à prévenir le MPP+ ?",
"position": 13,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, un mode de vie sain peut réduire le risque de maladies neurodégénératives."
}
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{
"@type": "Question",
"name": "L'éducation sur le MPP+ est-elle importante ?",
"position": 14,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, l'éducation sur les risques et les symptômes est essentielle pour la prévention."
}
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{
"@type": "Question",
"name": "Les contrôles réguliers sont-ils recommandés ?",
"position": 15,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, des contrôles réguliers peuvent aider à détecter précocement les symptômes."
}
},
{
"@type": "Question",
"name": "Quels traitements sont efficaces contre le MPP+ ?",
"position": 16,
"acceptedAnswer": {
"@type": "Answer",
"text": "Les traitements incluent des médicaments dopaminergiques et des thérapies physiques."
}
},
{
"@type": "Question",
"name": "La réhabilitation est-elle utile pour les patients exposés au MPP+ ?",
"position": 17,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, la réhabilitation aide à améliorer la mobilité et la qualité de vie des patients."
}
},
{
"@type": "Question",
"name": "Peut-on utiliser des antioxydants pour traiter le MPP+ ?",
"position": 18,
"acceptedAnswer": {
"@type": "Answer",
"text": "Des antioxydants peuvent être envisagés pour réduire le stress oxydatif lié au MPP+."
}
},
{
"@type": "Question",
"name": "Les thérapies complémentaires sont-elles bénéfiques ?",
"position": 19,
"acceptedAnswer": {
"@type": "Answer",
"text": "Certaines thérapies complémentaires, comme l'acupuncture, peuvent soulager les symptômes."
}
},
{
"@type": "Question",
"name": "Le traitement précoce du MPP+ est-il crucial ?",
"position": 20,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, un traitement précoce peut ralentir la progression des symptômes parkinsoniens."
}
},
{
"@type": "Question",
"name": "Quelles complications peuvent survenir avec le MPP+ ?",
"position": 21,
"acceptedAnswer": {
"@type": "Answer",
"text": "Des complications incluent des troubles moteurs sévères et des problèmes cognitifs."
}
},
{
"@type": "Question",
"name": "Le MPP+ peut-il entraîner des chutes ?",
"position": 22,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, les troubles de la marche augmentent le risque de chutes chez les patients."
}
},
{
"@type": "Question",
"name": "Y a-t-il un risque accru de dépression avec le MPP+ ?",
"position": 23,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, les patients exposés au MPP+ présentent un risque accru de dépression."
}
},
{
"@type": "Question",
"name": "Le MPP+ peut-il affecter la qualité de vie ?",
"position": 24,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, les symptômes parkinsoniens peuvent significativement réduire la qualité de vie."
}
},
{
"@type": "Question",
"name": "Des complications cardiovasculaires sont-elles possibles ?",
"position": 25,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, des études suggèrent un lien entre MPP+ et complications cardiovasculaires."
}
},
{
"@type": "Question",
"name": "Quels sont les principaux facteurs de risque du MPP+ ?",
"position": 26,
"acceptedAnswer": {
"@type": "Answer",
"text": "L'exposition à des pesticides et à des produits chimiques industriels augmente le risque."
}
},
{
"@type": "Question",
"name": "L'âge est-il un facteur de risque pour le MPP+ ?",
"position": 27,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, le risque d'effets néfastes du MPP+ augmente avec l'âge."
}
},
{
"@type": "Question",
"name": "Les antécédents familiaux influencent-ils le risque ?",
"position": 28,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, des antécédents familiaux de maladies neurodégénératives augmentent le risque."
}
},
{
"@type": "Question",
"name": "Le sexe joue-t-il un rôle dans le risque de MPP+ ?",
"position": 29,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, des études montrent que les hommes sont plus à risque que les femmes."
}
},
{
"@type": "Question",
"name": "Le mode de vie influence-t-il le risque de MPP+ ?",
"position": 30,
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"@type": "Answer",
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The development of orphan drugs (ODs) is challenging from both development and business perspectives because of their small patient populations. To overcome such business challenges, lifecycle managem...
The 203 ODs consisting of 173 active ingredients were approved in Japan between 2004 and 2019. Sixty-eight (39%) of the 173 active ingredients have additional indications, of which 57 have at least on...
Pharmaceutical companies were actively adding non-OD indications and were emphasizing the use of patent rights by registering extensions of substance or use patents for exclusive marketing periods. Th...
Out of 185 orphan medicinal products (OMPs) registered in 2015-2021, a mere 110 (59 percent) were available to Czech patients, and only 54 (29 percent) were officially reimbursed. Moreover, this propo...
The innovative legislation follows the recommendations for value assessment and funding processes for rare diseases and incorporates additional elements of value, such as the societal perspective. Fir...
We believe that this novel approach may offer satisfactory patient access to orphan drugs. Moreover, it serves as a real-world example of "value-based" decision making....
Rare diseases affect more than 30 million Americans. The passage of the Orphan Drug Act (ODA) in the United States in 1983 represented a launching point for a rare disease drug development revolution ...
Using an internal FDA database, we classified and analyzed all orphan drug designations and approvals from 1983 to 2022 by disease and therapeutic area....
Over the 40 years of the ODA, 6,340 orphan drug designations were granted, representing drug development for 1,079 rare diseases. Additionally, 882 of those designations resulted in at least one FDA a...
Researchers have estimated that there may be 7000-10,000 rare diseases that have been identified and described. Based on our study, we can conclude that around 5% of rare diseases have an FDA-approved...
In many countries, β-thalassemia (β-THAL) is not uncommon; however, it qualifies as a rare disease in the US and in European Union (EU), where thalassemia drugs are eligible for Orphan Drug Designatio...
This study aimed to identify different criteria for priority setting of rare diseases to help policy makers in making evidence-informed decisions....
A scoping review was conducted to comprehensively examine the existing various methods and criteria for prioritizing orphan drugs and rare diseases. We performed searching in Scopus, PubMed, Embase, a...
From the 1,580 identified publications, eleven articles were included. Multicriteria decision analysis was the most frequent method (seven out of eleven studies) used for priority setting. The extract...
Because of the high price of orphan drugs and limitations of using HTA for reimbursement of them, it is critical to explore them by precise technical methods like multiple criteria decision making in ...
Alpha-1 antitrypsin deficiency (AATD, OMIM #613490) is a rare metabolic disorder affecting lungs and liver. The purpose of this study is to assess the impact of the US orphan drug act on AATD by provi...
Rare diseases have been increasingly recognized as unmet medical and health needs worldwide; a growing demand for the development of orphan drugs emerges subsequently. Therefore, it is of great intere...
This study aims to reveal the current situation and trend of the clinical development of orphan drugs in China, based on the data collected from the Chinese official platform, dating from January 1, 2...
A total of 331 clinical trials for orphan drugs were extracted from the platform, covering 31 rare diseases and 124 drugs. Increases were seen in the annual number of clinical trials and drugs being t...
The growth trends illustrate the progress in clinical trial and drug development of rare diseases from 2013 to 2021. However, promoting orphan drugs development still is an important issue in China; a...
The Orphan Regulation ((EC) No 141/2000) has successfully redirected private and public investment towards previously neglected areas through incentives, regulatory obligations and rewards. However, t...
Expenditure on OMPs in Wales was analysed between the 2014/15 and 2019/20 financial years using data on prescriptions dispensed in primary care, secondary care, and specialised commissioned services. ...
Overall spend on OMPs and all medicines increased from £32 m to £82 m, and from £1,030 m to £1,198 m, respectively, with the proportion of spend on OMPs more than doubling from 3.1% to 6.9% per annum....
Both the number of OMPs and the amount spent on OMPs in Wales has increased over time, possibly as a consequence of favourable licensing conditions, permissive health technology assessment policies an...
This study aimed to establish a suitable value assessment framework for orphan medicinal products in China based on the multi-criteria decision analysis (MCDA) method....
First, a draft framework of the MCDA criteria was built based on a systematic literature evaluation and the EVIDEM framework tools. Second, stakeholder groups were formed and expert opinions were coll...
Combined with the stakeholder selection and suggestions in the stakeholder workshop, the framework criteria for the evaluation were constructed based on China's national conditions, which included 11 ...
MCDA is feasible for the value assessment of orphan drugs in China and can be used as a supplementary tool for drug access decisions in medical insurance. It is suggested to further improve the value ...
In the United States (US) and in Europe, cystic fibrosis (CF) qualifies as a rare disease, thus positioning the field to benefit from regulatory incentives provided by orphan drug designation (ODD) to...
We collected the products that received the ODD from the registries of the FDA and the EMA from 2000 to 2021, characterizing their nature, development stage, and type of sponsor. We categorized the st...
From 2000-2021, 107 ODDs were collectively granted by the FDA and the EMA for products developed for the treatment of CF. Although the trends of the number of ODDs granted remained stable over time, t...
Although ODDs for treating infection and other CF sequelae accounted for the majority, we observed a shift of ODDs toward mechanism-based products over the study period. In line with other rare diseas...
