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Nadine Dragin, researcher among an Inserm/UPMC/CNRS (French National Center for Scientific Research)/Institute of Myology team co-directed by Sonia Berrih-Aknin and Rozen le Panse from the Institute of Myology, based in the Pitié-Salpêtrière Hospital, AP-HP, brought to light the central role of AIRE, a key factor for immune tolerance, in the gender inequality regarding autoimmune diseases. … [Read more]
A research team from the Institute of Myology, led by Denis Furling, has taken part in a collaborative work coordinated by Nicolas Charlet (Institute of Genetics and Molecular and Cellular Biology, Strasbourg) and Masanori Takahashi (Osaka, Japan), which enabled to bring to light the key role of alteration (with expanded CTG repeats) of the splicing … [Read more]
Spring 2016 The Genethon International Postdoctoral Program 2016 is now open. Positions are intended for highly motivated scientists who wish to pursue their career in the research and development of therapies for rare diseases. Postdoctoral research positions are available for a period of three years (37-41 k€ annual gross income depending on qualifications and years … [Read more]
The congress Myology 2016, organised by AFM-Téléthon, is now coming to an end: over 900 international experts in the field (researchers, clinicians, doctors…) have compared their latest work for 5 days. This edition was undoubtedly marked by the beginning of the therapeutic era for neuromuscular diseases with the presentation of clinical trials results in human … [Read more]
The fourth day of Myology 2016, organised by AFM-Téléthon, opened on a plenary session dedicated to the different myology-related advances. H. Lee Sweeney from the University of Florida (USA), co-chairman of the congress, overviewed the trials using oligonucleotides in Duchenne muscular dystrophy. He underlined the need, for demonstrating the efficiency of products currently under clinical … [Read more]
Jerry Mendell told us about gene therapy in spinal muscular atrophy.
H. Lee Sweeney discusses the difficulty of having drugs approved for Duchenne Muscular Dystrophy.
Ichizo Nishino explains his successful results for Nonaka myopathy (GNE).
Wednesday opened on a symposium based on the development of innovative therapies, which make a new, booming type of medicine. Because the first drug candidates developed so far are only “prototypes”, researchers and doctors are building a strategy to tackle the disease on all fronts. Matthew Wood from Oxford University (UK) restored the expression of … [Read more]
Matthew Wood discusses the challenges of developing gene therapy