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CADAPERSEN
Generic Name: CADAPERSEN
ABSTRACT -
Cadapersen is a phosphorodiamidate morpholino oligomer (PMO) antisense therapy designed to induce exon skipping in the dystrophin pre-mRNA, restoring a truncated but partially functional dystrophin protein in Duchenne muscular dystrophy (DMD) patients amenable to specific exon skip. It is being evaluated in paediatric DMD patients in clinical trials.
DOSAGE -
Intravenous infusion once weekly; dose based on body weight (mg/kg) per clinical protocol.
SIDE EFFECTS -
- Infusion-related reactions
- Renal tubular dysfunction
- Headache
- Nausea
- Fatigue
ALLERGIC REACTIONS -
These symptoms may indicate an allergic reaction and require immediate medical attention.
- Hypersensitivity
- Urticaria
- Anaphylaxis (rare)
- Angioedema
DRUG INTERACTIONS-
- No significant CYP-mediated drug interactions expected (ASO metabolism)
- Concurrent nephrotoxic drugs increase renal risk
- Monitor renal function closely with aminoglycosides
PREAUTONS–
- Renal function monitoring mandatory — cystatin C and urine biomarkers
- Confirmed mutation amenable to exon skip must be documented
- Annual cardiac and pulmonary function assessments recommended
- Paediatric dosing weight-based with growth monitoring
WARNINGS –
- Renal toxicity including tubular dysfunction reported with PMO class
- Confirmatory dystrophin exon skip verified by muscle biopsy
- Not all DMD mutations are amenable to this therapeutic approach
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