To mark the day, ELA International launched an information campaign on leukodystrophies and a donation drive. All the ELA branches in Belgium, France, Germany, Italy, Luxembourg, Spain and Switzerland shared this campaign on social networks and in the press, supported by the broadcast of the Zidane, coach of researchers… spot.
Leukodystrophies are rare diseases
Leukodystrophies are rare genetic neurological diseases affecting the white matter of the nervous system, myelin. They result in the loss of all vital functions and cause severe physical and mental disabilities in children. Between 20 and 40 children in Europe are born with leukodystrophy, with symptoms appearing as early as the first year of life and progressively worsening as the child grows. Very few treatments are currently available for these rare diseases. Ongoing studies offer hope.
ELA International brings together researchers from all over the world
ELA International, which brings together researchers from all over the world, coordinates research into leukodystrophies and has chosen to concentrate its funding on programmes that are most applied to improving the state of health of patients. This objective can be achieved through studies/projects aimed at understanding the mechanisms of the disease, the natural history of the disease, in particular through the discovery of disease assessment tools and biomarkers, and the conduct of clinical trials.
ELA, une contribution à la recherche depuis 1992
Over the past twenty-nine years, ELA has contributed to the identification of rare diseases, the understanding of new mechanisms, the development of new-generation genome analysis methods and also a number of therapeutic revolutions such as haematopoietic stem cell transplants and gene therapy. Gene therapy is making progress and ELA has been promoting its development for several years. This therapeutic approach remains a real hope for patients suffering from certain forms of leukodystrophy.
Funding research during a pandemic
While the crisis has affected many of the research projects currently underway, some have been slowed or even suspended for several months. The pandemic has also had serious consequences, such as the postponement of certain clinical trials supported by ELA. This situation contrasts with the considerable sums invested in record time by governments and private laboratories to discover an anti-covid19 vaccine. In this difficult context, ELA is redoubling its vigilance and fighting spirit to continue to raise the profile of leukodystrophies internationally.
ELA’s families play an active role in research
With ELA, the mobilisation of patients and their families is unfailing, to contribute tò research into rare diseases, and is also part of a profound change in the relationship between doctors and patients by giving patients and their families a role-playing role via family/researcher meetings, international research symposia, Leuconnect…
