FAQs
How does Exondys 51 work to treat Duchenne muscular dystrophy?
Exondys 51 is an antisense oligonucleotide that binds to exon 51 of the dystrophin pre‑mRNA, causing the cellular machinery to skip that exon during processing. Skipping restores the gene’s reading frame, allowing production of a shortened but functional dystrophin protein.
Who is eligible for Exondys 51 therapy?
Only patients with a confirmed “exon 51‑amenable” mutation in the dystrophin gene, typically age 4 years or older, and who still have ambulatory or early‑stage disease can receive the treatment.
What are the common side effects of Exondys 51?
Most users experience mild infusion‑related reactions (fever, chills, rash) and may have transient changes in kidney function. Regular laboratory monitoring is required.
How much does Exondys 51 cost in the United States?
List prices range from about $300,000 to $350,000 per year. Insurance coverage varies, and many families rely on manufacturer assistance programs to offset out‑of‑pocket costs.
Can Exondys 51 be used together with other DMD medications?
Yes, it can be administered alongside standard supportive care such as corticosteroids and cardiac/respiratory therapies, but it should not be combined with another exon‑skipping agent.
