Neuromuscular diseases program

Among all the types of rare diseases, Genethon has chosen to focus its efforts on Neuromuscular disorders, which are the key focus of AFM and its spearhead organizations.

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Research is currently underway in Duchenne muscular dystrophy (skeletal muscle and heart function), spinal muscular atrophies (SMA), limb girdle muscular dystrophies and myotubular myopathies.

Genethon is especially concentrating its efforts on developing modes of administration that ensure easy and effective distribution of the therapeutic gene-drug to a wide range of muscles, as well as on large-scale bioproduction of AAV (adeno-associated virus) vectors.