A growing number of infants diagnosed with spinal muscular atrophy (type I or type II) are benefiting from adeno-associated virus (AAV)-mediated gene therapy. Clinicians at the Necker-Enfants Malades hospital report on their respiratory progress:
- 15 infants (including one case of SMA type II) with a median age of 8.6 months (3.8 to 12.6 months) were included in the study and followed for at least 24 months,
- most respiratory parameters, including those from polysomnographic studies, were normal at inclusion and at the end of the study,
- only three children required non-invasive ventilation.
The authors emphasize the need to continue to monitor the respiratory status of these children, even though treatment appears to have normalized or stabilized ventilatory function.
