“Axel is walking with a walker, pulling himself up on everything and opening every drawer. He’s already 13 months old, and without the treatment we would be halfway through Axel’s life - and that wouldn’t have been much of a life either,” Melitta shared.

The first few days of a child’s life are usually filled with joy, excitement, and an imagination full of endless possibilities. But for Axel’s parents, that was overshadowed by fear. Just days after welcoming their newborn, they were faced with a devastating diagnosis – spinal muscular atrophy (SMA).

SMA is a rare genetic condition that affects the motor neurons of the spinal cord, causing muscle weakness, damage, wasting (atrophy) and eventual loss of function. This disease, if left untreated, is the leading cause of death for children under the age of two. Gene therapy is giving Axel a newfound hope for the future.