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MUSCULO-SKELETAL SYSTEM › OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM › OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM › Other drugs for disorders of the musculo-skeletal system
aceneuramic acid
M09AX05
Forms & Strengths
- Extended-release floating tablets: 1 g
Adult Dosing
- 3.5 g orally three times daily with meals
Pediatric Dosing
- Safety and efficacy in pediatric patients have not been established
Indications
- Treatment of GNE myopathy (hereditary inclusion body myopathy)
Mechanism of Action
- Acts as a substrate replacement therapy to restore sialic acid synthesis in patients with GNE myopathy caused by UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine kinase deficiency
Contraindications
- Hypersensitivity to aceneuramic acid or any of its excipients
Adverse Reactions
- Diarrhea
- Nausea
- Headache
- Abdominal pain
- Flatulence
- Nasopharyngitis
Drug Interactions
- No formal clinical drug-drug interaction studies have been conducted
- Renally eliminated drugs may have theoretical competition for renal clearance pathways, though clinically significant interactions are not well documented
Curated Content: Needs Vetting Before Put to Clinical Use