Zolgensma for Spinal Muscular Atrophy
Pediatric Neuromuscular Clinic
American Family Children's Hospital
1675 Highland Ave.
Madison, WI 53792
Zolgensma is the new gene therapy that prevents further motor neuron and muscle degeneration by replacing the defective or missing gene (SMN1) that causes Spinal Muscular Atrophy (SMA). The FDA announced its decision about this drug on May 25, 2019.
How does Zolgensma gene therapy work?
Zolgensma is a gene therapy designed to replace the function of the nonworking or missing gene that causes SMA. Zolgensma works by using a new gene to tell motor neuron cells to produce more survival motor neuron (SMN) protein. Motor neuron cells need SMN protein to survive and support muscle functions.
Who is eligible for Zolgensma?
Zolgensma gene therapy will be available for the treatment of children with SMA less than 2 years of age. Eligible patients cannot have antibodies to the virus, AAV9 or adeno-associated virus 9 as AAV9 is an important part of how Zolgensma works as gene therapy. These restrictions are based on the population and dose approved by the FDA.
- Health care staff provide Zolgensma gene therapy through an IV that lasts 60 minutes. Only one dose is needed.
- This treatment sends a healthy copy of the SMN1 gene to cells throughout the body and reach the cells in the spinal cord
- When the healthy SMN1 gene reaches a child's cells, it begins producing normally-functioning SMN protein, helping improve motor neuron function and increasing the chance for survival
Does UW Health Offer Zolgensma?
UW Health is happy that the Zolgensma gene therapy has been approved and excited about the potential benefit to patients with spinal muscular atrophy (or SMA) who are under 2 years of age and without antibodies to the virus, AAV9. While we understand the urgency of treatment, we are exploring our ability to offer the treatment and considering arrangements including working with insurance companies. As soon as any additional information is available, we will share it with families of SMA patients that meet Zolgensma criteria.