Gene therapy has become a treatment option for many infants with severe forms of spinal muscular atrophy (SMA) and tomorrow for those diagnosed at a presymptomatic stage through newborn screening. Polish researchers have been looking at the practical consequences of such a therapeutic approach on how to vaccinate these children.
- There are no international recommendations in this area.
- However, children treated with gene therapy receive prolonged corticosteroid therapy (at least one month, sometimes much longer) in order to avoid an immune reaction and/or liver toxicity.
- The Polish Vaccination Committee has issued its first recommendations in this area, which could become standard practice elsewhere in the world.
- Of particular concern are measures to protect against tuberculosis, as BCG is usually given in the first few months of life.
