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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
nusinersen
M09AX07
Forms & Strengths
- Intrathecal injection: 12 mg/5 mL (2.4 mg/mL) in a single-dose vial
Adult Dosing
- 12 mg (5 mL) administered intrathecally every 4 months after the loading period
Pediatric Dosing
- Loading dose: 12 mg intrathecally every 14 days for 3 doses
- Fourth loading dose: 12 mg 30 days after the third dose
- Maintenance dose: 12 mg intrathecally once every 4 months
Indications
- Treatment of spinal muscular atrophy (SMA) in pediatric and adult patients
Mechanism of Action
- Antisense oligonucleotide (ASO) designed to alter the splicing of SMN2 pre-mRNA in order to increase production of full-length SMN protein
Contraindications
- History of hypersensitivity to nusinersen or any of its excipients
Adverse Reactions
- Lower respiratory infection
- Constipation
- Teething
- Vomiting
- Back pain
- Headache
- Post-lumbar puncture syndrome
- Thrombocytopenia
- Renal toxicity
Drug Interactions
- Formal clinical drug interaction studies have not been performed
- Potential for nephrotoxicity and coagulation abnormalities requires monitoring when used with other potentially nephrotoxic or antiplatelet/anticoagulant agents
Curated Content: Needs Vetting Before Put to Clinical Use