After great progress in the survival of patients, research has tackled a challenge: repairing the gene causing the disease.
“We are really living a period of revolution. For a long time, we explained to families: gene therapy is the great hope, the future… Now we are there. Until now, the symptoms of the disease have been treated. Now we can treat its cause ”, rejoices the Pr Vincent Laugel, pediatric neurologist at the Strasbourg University Hospital. The success of the treatment by gene therapy of another rare disease whose prognosis it revolutionized, spinal muscular atrophy (SMA), pave the way: a single injection of Zolgensma is enough to cure children (around 120 per year in France) formerly doomed.
Rare genetic disease, Duchenne muscular dystrophy (or Duchenne muscular dystrophy, DMD), the most common genetic neuromuscular disease, affects approximately 1 in 3,300 boys and almost no girls. It is due to the mutation of a gene on the X chromosome encoding a protein, dystrophin, essential for the functioning of muscles. Located under the cell membrane
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