Cecilia Pessoa Gingerich

This is the first gene therapy approved for a devastating condition that leads to permanent ventilation or death for many patients by age 2.

Crizanlizumab is being developed to prevent painful and unpredictable vaso-occlusive crises in patients with sickle cell disease.

The FDA has accepted a Biologics License Application as well as granted Priority Review for the gene therapy that treats spinal muscular atrophy (SMA) Type 1.

Children receiving AVXS-101 have shown such improvement that researchers are calling for an update to the CHOP-INTEND scale used to track their progress.