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La prise en charge thérapeutique du syndrome hémolytique urémique atypique chez l'adulte et l'enfant et ses futures évolutions possibles en France
Mémoire de Master / Thèse d'exercice

La prise en charge thérapeutique du syndrome hémolytique urémique atypique chez l'adulte et l'enfant et ses futures évolutions possibles en France

Pauline Stefanello
Masters , Université de Montpellier
06/09/2024

Résumé

Syndrome hémolytique et urémique -- Thérapeutique Eculizumab Essais cliniques des médicaments -- France Médicaments en essais cliniques
Atypical hemolytic uremic syndrome is a rare disease, not well known to the public at large. In France, only one molecule is covered by Social security, and few alternatives exist anywhere in the world. It is therefore important to develop new treatments to make this disease accessible to everyone, in every country. Eculizumab has some undesirable side effects and disadvantages concerning its route of administration. A number of pharmaceutical companies have begun to take an interest in this disease and have launched clinical trials. These include Roche with crovalimab, Novartis with iptacopan, Omeros with narsoplimab, Alnylam with cemdisiran and NovelMed with NM8074. Many molecules are currently being developed with new mechanisms of action and new modes of administration, which could be a major step forward for patients suffering from HUS atypical and their care.

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