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Therapies
Sous presse. Epreuves corrigées par l'auteur. Disponible en ligne depuis le dimanche 1 mars 2020
Doi : 10.1016/j.therap.2020.02.009
Received : 16 September 2019 ;  accepted : 15 November 2019
An old drug for a rare disease: The example of celiprolol for vascular Ehlers-Danlos syndrome
 

Pierre Boutouyrie
 Service de pharmacologie, INSERM U970, hôpital européen Georges-Pompidou, université de Paris, Assitance publique–Hôpitaux de Paris, 20, rue Leblanc, 75015 Paris, France 

Summary

Vascular Ehlers-Danlos syndrome (OMIM 130050, 1/150,000 birth) is caused by mutations in collagen 3A1  gene. It is associated with severe phenotype associating early arterial dissection and rupture, digestive and uterine perforations, and skin and joints fragility. Until recently, no treatment was available. Celiprolol, a beta1 antagonist with beta2 partial antagonist properties betablocker was tested in a randomized, controlled trial. We could show that this compound was associated with a 3-fold decrease in major events related to the disease. This effect was similar in molecular-proven patients. Administration of celiprolol in a cohort of patients followed routinely in France was accompanied to similar benefit. Celiprolol is unavailable in the USA. The ACER Therapeutics company applied for new drug application (NDA) to the Food and Drug Administration.

The full text of this article is available in PDF format.

Keywords : Vascular Ehlers-Danlos syndrome, Celiprolol, Randomized controlled trial




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