FDA Approves Zolgensma for SMA Treatment
This is the first gene therapy approved for a devastating condition that leads to permanent ventilation or death for many patients by age 2.
FDA Grants Breakthrough Therapy to Sickle Cell Treatment Crizanlizumab
Crizanlizumab is being developed to prevent painful and unpredictable vaso-occlusive crises in patients with sickle cell disease.
FDA Accepts Filing for Zolgensma for Spinal Muscular Atrophy Type 1
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 with SMA1 Show Remarkable Progress on Gene Therapy
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.