Miscellaneous musculoskeletal disorders:

Indications for EXONDYS 51:

Duchenne muscular dystrophy (DMD) in patients with a confirmed DMD gene mutation that is amenable to exon 51 skipping.

Adults and Children:

May apply topical anesthetic cream to IV site prior to administration. Give by IV infusion over 35–60mins. 30mg/kg once weekly.


Treat appropriately and consider slowing the infusion or interrupting therapy if a hypersensitivity reaction occurs. Renal impairment. Pregnancy. Nursing mothers.

Pharmacologic Class:

Antisense oligonucleotide.


Do not mix or infuse with other medications concomitantly via same IV access line.

Adverse Reactions:

Balance disorder, vomiting, contact dermatitis; hypersensitivity reactions.

Generic Availability:


How Supplied:

Single-dose vial (2mL, 10mL)—1