Monthly Archives: July 2017

Monday 31st july 2017

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Friday 28 july

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Thursday 27 july

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Wednesday 26 july

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Microdystrophin restores muscle strength in Duchenne muscular dystrophy

Researchers from Généthon, the AFM-Téléthon laboratory, Inserm (UMR 1089, Nantes) and the University of London (Royal Holloway) demonstrated the efficacy of an innovative gene therapy in the treatment of Duchenne muscular dystrophy. Indeed, after injecting microdystrophin (a “shortened” version of the dystrophin gene) via a drug vector, the researchers managed to restore muscle strength and stabilise the clinical symptoms in dogs naturally affected by Duchenne muscular dystrophy. A first. This work, published today in Nature Communications, has been achieved thanks to donations from the French Téléthon.

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Tuesday 25 july

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Monday 24 july

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Friday 21st July 2017

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Gene therapy: first results in children with Sanfilippo B syndrome

On July 13, 2017, the journal Lancet Neurology published the results of a gene therapy trial conducted in four children with Sanfilippo type B syndrome (also known as MPS IIIB). This trial is the achievement of a two-decade partnership with financial support of AFM-Téléthon and the cooperation of the charity “Vaincre les Maladies Lysosomales” (VML). After monitoring of the treated children for 30 months, Dr. Jean-Michel Heard, from the Institut Pasteur and Inserm, and Professors Marc Tardieu and Michel Zérah, from the Paris public hospital administration (AP-HP) and the Paris-Sud and Paris Descartes Universities, conclude that the treatment was well tolerated and associated with neurocognitive benefits for the patients.

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Wednesday 19th July 2017

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