As therapeutic options for Duchenne advance, we are seeing continued development of additional AAV gene transfer therapies. There is currently one FDA approved gene therapy, ELEVIDYS, and several more in clinical trials.
With all therapies, there are associated side effects and it’s critical that families and individuals who receive gene therapy (or any therapies) understand the signs and symptoms of a potential emergency and when to seek care.
PPMD has created a Post AAV Gene Transfer Therapy Emergency Card as a supplemental resource to the information provided by your/your child’s care team. This card includes critical information for both families and healthcare providers to know what to look for and monitor during potential emergencies after receiving an AAV gene therapy.
This Emergency Card acts as a resource for emergency personnel while your primary neuromuscular or gene team can be contacted for specific treatment management. It is intended to provide interim guidance on labs and imaging to help understand if a potentially related side effect is occurring.
At PPMD, we want every family to be prepared and equipped with resources for any situation that may occur. If you are ever unsure of your child’s symptoms, do not wait! Always contact your medical team right away for guidance on next steps or go to the emergency department.
You can order your Post AAV Gene Transfer Therapy Emergency Card here.
Find more information on post gene therapy emergency care here.
Find a list of PPMD’s CDCC After-Hours Numbers here.
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