Yesterday the board of directors at Genethon nominated a new president to head the laboratory created in 1990 by the AFM (French Muscular Dystrophy Association). Thus Yves Champey succeeds Bernard Barataud, who has decided to hand over the reins after heading the laboratory between 1990 and 1995 and then between 1999 and 2008.

Five years after identification of the gene responsible, a team of Spanish and French researchers (Carlos Lopez-Otin and coll. - University of Oviedo - and Nicolas Lévy and coll. - Inserm/AP-HM, Marseille, France) has validated in the mouse a treatment for progeria, a rare disease causing accelerated and premature ageing. This treatment, combining two existing pharmacological molecules, should slow down the progression of this disease which has hitherto
remained untreatable.
Subject to the authorisation of health agencies, this treatment could very soon be tested on 15 children in Europe. This represents a major advance for the families affected by this disease and – more generally – for the understanding of the normal ageing process.
From identification of the gene to the development of treatment and the projected clinical protocol, this work – published in the review Nature Medicine – has received the financial backing of the Association Française contre les Myopathies (Muscular Dystrophy
Association) thanks to French Telethon donations.

Since 2000 each International Congress of Myology organised by the AFM has marked a step in the research concerning neuromuscular diseases. This third congress marked that of clinical development – many of the interventions during Myology 2008 were oriented towards therapeutic prospects or presented trials in humans. There is a boom in stem cells, particularly for the heart; gene surgery, which is being enriched by various techniques; as well as pharmacology etc. The time has arrived for treatments and with it, the development of evaluation methods for clinical trials has become of paramount importance. This is why Myology 2008 shared its programme this year with the 5th International Rehabilitation Congress in Neuromuscular Diseases. Through these two congresses the AFM has demonstrated that – from laboratory to patient – now is the time of the turning point.

For its last day, Myology 2008 shared its programme with the opening of the 5th international rehabilitation conference in neuromuscular diseases. At the crossroads of research and medicine, the two congresses drew up an inventory this morning on the latest innovations in the matter of gene-based therapies.