TREAT-NMD partners

Created in 1958 and managed by a group of patients and their families, AFM has become a strong organisation combining the militant energy of its founding members, the professionalization of its volunteer managers and the commitment of its employees. AFM is involved in the development of scientific tools for the study of genetic and rare diseases in general, and provides scientists with new means and knowledge to accelerate understanding of genetic diseases and open treatment avenues based on an understanding of the genes.

The University of Basel has a long history of excellence in Life Sciences. In particular, the Biozentrum, founded in 1971, was among the first institutions world-wide dedicated to molecular biology.

The European Organisation for Rare Diseases (EURORDIS) is a non-governmental federation of nearly 480 rare disease organisations in 45 different countries (of which 25 are EU member states), covering over 1,200 rare diseases. It is therefore the voice of 30 million patients affected by rare diseases throughout Europe.

Since 1990, Telethon research aims at moving towards goals that get closer and closer to achieving cures for inherithed neuromuscular diseases and other genetic disorders.

It does this by relying on a group of internationally renowned scientists that make up the Telethon medical-scientific Committee, in choosing the worthiest projects. Alongside scientific qualities, the Committee uses another two other important criteria in evaluating proposed projects: their relevance to Telethon's mission, and their closeness to a cure, that is: those projects with a greater possibility of generating information useful to developing or improving a therapy.

The Friedrich-Baur-Institute is part of the Medical Faculty of the Ludwig-Maximilians University in Munich. In association with the Neurological Clinic, the Institute is dedicated to patient care and excellence in the field of neuromuscular disorders.

First a pioneer of human genome research, Genethon now explores the new therapeutic hypotheses stemming from the extraordinary progresses of contemporary biology.

GenoSafe is a service company specialised in the evaluation of the efficacy and safety of biotherapeutic products (viral and non-viral vectors, vaccines, recombinant proteins, monoclonal antibodies, cellular products …). 

Since the sequence of the human genome is largely known, allowing the identification and characterization of all human genes, the next era of human genetics will be marked by genome-wide analyses in both monogenetic and multifactorial disorders, as well as functional studies aiming at the understanding of complex interactions between multiple genes.

The department is a Reference diagnostic laboratory in France for various diseases including including several neuromuscular diseases DMD, BMD and DMC1A. The laboratory has a great expertise in the detection of molecular defects at the DNA and RNA level, using state-of-the-art technologies, and is particularly experienced in the field of splicing defects and RNA analysis. In addition, this laboratory is one of the three authorized for pre-implantation genetic diagnosis (PGD) in France, and developed a strong PGD activity on several muscular diseases including DMD and DM1.

The Institute of Myology was initiated by a group of leaders, patients and families from the French Muscular Dystrophy Association (Association Française contre les Myopathies, AFM) who wished to establish a point of reference where specialised consultations, fundamental and clinical research teams and teaching on muscle and muscle diseases would all be grouped together in a public hospital.

Karolinska Institute is a longstanding national centre for neuromuscular disorders, involving basic science and clinical research.

King's College London is currently the largest centre for the education of healthcare professionals in Europe, with more Medical Research Council Centres than any other.

At the Leiden University Medical Centre (LUMC), we are continually seeking to improve the quality of healthcare. The LUMC aims for excellence in patient care, research, teaching, training and continuing education and has a long history in the field of neuromuscular disorders.

The Department of Molecular Genetics and Diagnostics at the National Institute of Environmental Health in Hungary is specialised in the DNA diagnosis of neuromuscular disorders.

The Newcastle Muscle Centre is part of the Institute of Genetic Medicine, a 5* research organisation within Newcastle University. It is a centre of excellence for diagnosis, management and research into inherited neuromuscular diseases.

NOVAMEN, member of ACIES Consulting Group, specializes in collaborative research management, accompanies private and public stakeholders in European research in the strategic and operational management of their collaborative research projects, with a particular focus on European Union Framework Projects.

Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focusing on the development and marketing of innovative pharmaceutical products for the treatment of orphan neuromuscular diseases. Santhera currently has three compounds in seven clinical-stage development programs.

Established in 1989, the ENMC has been instrumental in facilitating communication and support for the European neuromuscular disease community.

The MD-NET was established in 2003 as an interdisciplinary network bringing together experts from different areas to collaborate on muscular dystrophy research.

The mission of the Functional Genomics Unit (FGU) is to use genomic information to determine mechanisms of disease in order to develop novel therapeutic approaches. In the FGU we are combining the power of computational analyses and the latest experimental technologies in model organisms to reveal the roles of genes and genomes in health and disease.

The Universitat Autònoma de Barcelona plays a leading role in scientific research in Spain. Its main areas of investigation include Biotechnology and Biomedicine, as well as a wide range of disciplines all integrated into one area to promote multidisciplinarity in research. The university coordinates a potent scientific and technological centre (the Esfera UAB), promotes new business projects, and directs and projects its productive activity towards its socio-economic surroundings by disseminating new knowledge.

The UCL partnership comprises the 6 Belgian Neuromuscular Reference Centres, located at the University Hospitals of Louvain, Liège, Leuven, Gent, Brussel, and Antwerpen. One of the goals of extending the UCL partnership to these 6 national Belgian Neuromuscular Reference Centres is to facilitate patient registration in national and supranational registries (TNMD) (WP04).

The Neuromuscular Centre at the Institute of Child Health has a longstanding tradition of excellence in the field of diagnosis, management and care of childhood neuromuscular conditions. The strength of the unit is the large critical mass of patients, the diagnostic pathology expertise together with an active program of research and key national and international collaborations.

The TREAT-NMD Clinical Trials Coordination Centre (CTCC) has been set up to work with pharmaceutical companies to facilitate every aspect of the trials process in Europe.