4 Iconography
Access to the text (HTML) Access to the text (HTML)
PDF Access to the PDF text

Access to the full text of this article requires a subscription.
  • If you are a subscriber, please sign in 'My Account' at the top right of the screen.

  • If you want to subscribe to this journal, see our rates


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 


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

© 2020  Société française de pharmacologie et de thérapeutique@@#104156@@
EM-CONSULTE.COM is registrered at the CNIL, déclaration n° 1286925.
As per the Law relating to information storage and personal integrity, you have the right to oppose (art 26 of that law), access (art 34 of that law) and rectify (art 36 of that law) your personal data. You may thus request that your data, should it be inaccurate, incomplete, unclear, outdated, not be used or stored, be corrected, clarified, updated or deleted.
Personal information regarding our website's visitors, including their identity, is confidential.
The owners of this website hereby guarantee to respect the legal confidentiality conditions, applicable in France, and not to disclose this data to third parties.
Article Outline