AFM Scientific Press Conference: First results of innovative therapies for rare diseases for the benefit of everyone

During a press conference that was held on November 18, 2010 at the Institute of Myology, Paris, AFM (French Association against Myopathie) summarized the first results which have been obtained thanks to innovative therapies (gene or cell therapy) used for rare disorders.

Laurence Tiennot-Herment, President of AFM and Genethon and Frederic Revah, Managing Director of Genethon were accompanied by 4 experts:

  • Prof. Adrian Thrascher, Great Ormond Street Hospital in London
  • Prof. Marc Peschansdi, Head of I-Stem, Institute of stem cells for the treatment and study of monogenic disorders (Evry)
  • Prof. Marina Cavazzana-Calvo, Department of Biotherapies and the Pediatric Immunology and Hematology Unit, Hopital Necker Enfants Malades, Paris.
  • Dr. Nicolas Ferry, Gene Therapy Laboratory, INSERM ERM 0105, Hotel Dieu University Hospital, Nantes.

This was also an occasion for clinicians and scientists to emphasize that these treatments may also be useful for common disorders. More than ever, research supported thanks to gifts to the Telethon is synonymous with benefits for all.

Furthermore, as Frederic Revah underlined during this press conference, “if AFM has no more money, some clinical trials will be halted. We will then have to explain to the patients that we have potential treatments, but they will not be able to take advantage of them.”