In case you missed it, PPMD was joined by Drs. Russ Butterfield of University of Utah and Sue Apkon of Colorado Children’s earlier this week to talk through some of the common questions around the recent FDA accelerated approval of ELEVIDYS. 

During the webinar, Drs. Butterfield and Apkon provided an overview of how gene therapy works, eligibility criteria listed in the ELEVIDYS label, risks and benefits, as well as logistics for accessing therapy safely. We were assured clinics are working hard to ensure pathways are in place to administer this treatment as quickly as possible, but this does require much preparation and communication among hospital/health system administration, pharmacies, and insurers.

Each patient and circumstance is unique, and it’s important to speak to your neuromuscular provider about your child’s potential eligibility for receiving ELEVIDYS and the process for receiving treatment. Visit PPMD’s FAQ for more information about ELEVIDYS.

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If you have questions about your child’s genetic mutation and potential eligibility for ELEVIDYS for other Duchenne specific treatments or trials, you can schedule a 1:1 appointment with one of PPMD’s genetic counselors here or email coordinator@parentprojectmd.org.

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