31 May 2012

Pooling expertise on rare diseases

Illustration for article: Pooling expertise on rare diseases
To accelerate research for rare diseases, it is essential to pool knowledge and expertise on an international scale. This is one of the working themes of AFM-Téléthon.

Our research crosses borders

Qualifying a disease as rare means that patients are also rare, with some pathologies affecting no more than a hundred patients throughout the world. Consequence: knowledge of these diseases is often fragmented, with few experts and it is difficult to constitute the patient cohorts needed for drug trials. Whether developing new treatments or improving patient care, international cooperation is essential. AFM-Téléthon encourages this.

European networks

For example, the European network Treat-NMD was formed at the association's initiative in 2007 to accelerate the development of innovative treatments for neuromuscular diseases. Funded by the European Union, it includes twenty-four partners (researchers, physicians, industrialists, patient associations) from eleven countries and has already provided the scientific and medical sectors with several tools such as a European database for all neuromuscular pathologies, a network of DNA, cell and tissue banks (Eurobiobank) and care standards for infantile spinal muscular atrophy and Duchenne muscular dystrophy. Similarly, AFM-Téléthon supported MyoRes, a network of fundamental research into muscle biology, a programme which ended in 2009 and helped place Europe at the forefront of this field.

AFM-Téléthon also develops links with other patient associations throughout the world. For example, it is a member of SMA-Europe which launched a call for bids in 2009 to fund international research projects on spinal muscular atrophy.