Important considerations
It’s normal to have questions about gene therapy. Read on to learn about how gene therapies are administered, what treatment may look like, answers to common questions, eligibility, and potential risks.
Pfizer's gene therapy for Duchenne muscular dystrophy is being evaluated in clinical trials and is not approved by the FDA. If FDA approved, it may not be an appropriate treatment for all children with Duchenne.
How gene therapies may be administered
Gene therapies can be given as in-vivo or ex-vivo.1 In-vivo gene therapy is given by intravenous (IV) infusions. This means it goes directly into the bloodstream.1 Ex-vivo gene therapy removes selected cells from the patient’s body for treatment. These cells are then returned into the body.1
In both cases, gene therapy is given at an infusion center.2 These centers are where patients receive gene therapies and may be monitored after treatment. Gene therapies are often given by IV infusions.1 This type of infusion helps introduce the medicine into the body through a needle or small tube.
Eligibility
Not all gene therapy treatments will be right for everyone. A child may not qualify for a treatment based on their age, the stage of their condition, if they have any other medical conditions, or if they have neutralizing antibodies (NAbs).3,4 NAbs may attack and destroy the vectors that deliver gene therapy.5
Each gene therapy option will have different eligibility criteria and may not be right for everyone. Work together with your care team to find an appropriate treatment plan.
Potential
risks
FAQs
Who can receive gene therapy?
Not all gene therapy treatments are right for everyone. A child may not qualify for a treatment based on their age, the stage of their condition, if they have any other medical conditions, or if they have neutralizing antibodies (NAbs).3,4 If gene therapy is not the right option, work together with your care team to find an appropriate treatment plan.
What are potential risks of gene therapy?
There are possible serious risks with gene therapy.6 These risks may include the development of an immune reaction after treatment.6,7 Studies are ongoing to understand the potential risks and benefits of gene therapy and families of children with Duchenne should always talk with their care team before making any treatment decisions.6
How long does gene therapy last?
Clinical trials are helping us learn more about gene therapy, such as how long the potential benefits may last. It’s important to learn everything you can about gene therapy in partnership with your child's care team. This includes the potential risks, benefits, and long-term follow-up.8
A look inside the potential gene therapy process
The gene therapy process can be different for every family. Here's what the process could look like—and don't forget, if you are considering treatment for your child, your care team will help along the way.
Pre-infusion
First, your child's care team will run a number of tests to see if gene therapy could be an option. If so, the care team schedules the infusion and uses the time in between to prepare you and your child for treatment.
Together with your care team, you and your child should make preparations for the infusion day itself and for potential monitoring to follow.
Infusion day
You and your child will arrive at the infusion center, where the care team administers the infusion. How long this process may take depends on the type of treatment.
Consider asking the care team about any additional support you and your child may need on infusion day.
Image is not an actual representation of the infusion process.
Post-infusion
Immediately after treatment, you and your child may need to stay close by for monitoring. The care team will determine how long your child will need to be monitored and how often they need to follow up with your child to track their progress.
Resources
Explore resources that can help you discuss Duchenne and gene therapy with your care team, plus find links to helpful support groups.