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(e TEP lir sen)

Index Terms

  • Exondys 51

Dosage Forms

Excipient information presented when available (limited, particularly for generics); consult specific product labeling.

Solution, Intravenous [preservative free]:

Exondys 51: 50 mg/mL (2 mL, 10 mL)

Brand Names: U.S.

  • Exondys 51

Pharmacologic Category

  • Antisense Oligonucleotide

Use: Labeled Indications

Duchenne muscular dystrophy: Treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.


There are no contraindications listed in the manufacturer's labeling.

Adverse Reactions

Incidences include reactions experienced with approved and unapproved doses.


Central nervous system: Equilibrium disturbance (38%)

Dermatologic: Contact dermatitis (25%), excoriation (≥10%), skin rash (≥10%)

Gastrointestinal: Vomiting (38%)

Hematologic & oncologic: Bruise (≥10%)

Local: Catheter pain (≥10%)

Neuromuscular & skeletal: Arthralgia (≥10%)

Respiratory: Upper respiratory tract infection (≥10%)

<1% (Limited to important or life-threatening): Facial flushing (transient; on infusion day), flushing (on infusion day), increased body temperature (transient; on infusion day)