Reference centers

In the context of the "National Plan for Rare Diseases 2005-2008,” the Ministry of Health identified the well-known international centers of expertise, labelled as reference centers for a rare disease or for a group of rare diseases.

The contribution of these medical centers for rare diseases is essential to the improvement of the quality of life for patients and their close ones. Their mission is, in fact, to facilitate diagnosis and to define the therapeutic and psychological course of treatment as well as the social support needed by the patients and their families.

18 reference centers for rare genetic children’s diseases have been accredited at the Necker-Enfants malades Hospital thanks to the excellence of the researchers and practitioners present on site.

As such, these 18 centers have received annual financing in the amount of 4.3 million Euros to coordinate their activity nationwide and, more often, worldwide.

 

Reference Centers for Rare Diseases at Necker Children’s Hospital :

Of the hundred existing nationwide reference centers for rare diseases, 18 have been created on the site of Necker-Enfants malades Hospital and are associated with the Imagine Foundation :


Genetic Diseases of a cutaneous nature
Professor C. Bodemer

Rare digestives intestinal diseases
Professor O. Goulet

Hereditary diseases of the metabolism
Professor P. de Lonlay

Mitochondrial Diseases CARAMMEL
Professor A. Munnich

Rare Epilepsies
Professor O. Dulac

Hereditary child and adult renal diseases
Professor C. Antignac et Professeur R. Salomon

Hereditary immunitary deficits
Professor A. Fischer

Juveniles arthritis
Professor P. Quartier

Complex cardiac malformations
Professor D. Bonnet

Rare diseases in ophthalmology
Professor J.-L. Dufier

Mastocytosis
Professor O. Hermine

Cranio-facial Dysostosis
Professor C . Sainte-Rose

Major sickle-cell syndromes
Professor M. de Montalembert

Ondine’s Syndrome
J. Amiel

Constitutional bone diseases
Professor M. Le Merrer

Neuromuscular Diseases
I. Desguerre

Competencies relating to Cystic fibrosis
G. Lenoir

Embryonic developmental anomalies of genetic origin
Professor S. Lyonnet

Non-syndromic fixed mental deficiencies of genetic origin

Ano-rectal and pelviennes deformations
CEMARA database

The CEMARA program exists to help Public Health authorities in their decision-making process relating to rare diseases. It is based on the storage and use of data present on computer networks. CEMARA was conceived and developed around a computer architecture of the biostatistical and computer laboratory of the Necker-Enfants maladies Hospital group. It forms a platform dedicated to epidemiology and support of clinical research in rare diseases.


This program has for objective to:
  • Contribute to the missions of the Center for Rare Diseases (CMR).
  • Count and characterize patients undergoing treatment for rare diseases in France and its dependencies.
  • Describe the “rare diseases” activity of the reference and competency centers.
  • Inform about the evolving tendencies in health care needs of the population and of treatment practices.
  • Contribute to help Public health authorities in their decision-making process relating to adaptation between offer and needs of care.

A strong link with Orphanet and Genatlas

CEMARA shares a common definition of diseases with Orphanet and localizes and identifies the disease genes with Genatlas.

A network with nationwide coverage

49 Rare Disease Centers brought together : 33 centers presently active and 14 centers to be integrated in 2009.

More than 55,000 patients registered in two years, since May 2007.


Site address : https://cemara.org/presentation/show.jsp

A password given to members of the Rare Diseases Center is needed to access the site.