MEDONEFARMA
ETEPLIRSEN LIQUID
PRODUCT DESCRIPTION
Eteplirsen is an antisense oligonucleotide used to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.
CHEMICAL NAME
5′-[P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylphosphonamidate] [P-deoxy-P-(dimethylamino)](2′,3′-dideoxy-2′,3′-imino-2′,3′-seco)(2′a→ 5′)(C-m5U- C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G) RNA.
Brand Names
Exondys 51
Category
Neuromuscular / Duchenne Muscular Dystrophy (Injection)
DOSAGE FORM
Injection: 50mg per ml single dose vial Intravenous use.
INDICATIONS
Eteplirsen injection is used to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the dystrophin gene amenable to exon 51 skipping. DMD is a rare, inherited muscle disease that is caused by absence of a protein called dystrophin, which helps keep muscle cells intact.
NDC CODE
60923-363-02
STORAGE & SHELF LIFE
- Shelf life: 1.5 years (18 months)
- Store at2°c to 8°C (36°F to 46°F).
Anti-infectives
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