Formulary
Ataluren: Indications, Dosing, Side Effects and Interactions
Ataluren restores the synthesis of dystrophin by allowing ribosomes to read through premature stop codons that cause incomplete dystrophin synthesis in...
MedNext Academy | 3 min read
Ataluren: Indications, Dosing, Side Effects and Interactions
Ataluren restores the synthesis of dystrophin by allowing ribosomes to read through premature stop codons that cause incomplete dystrophin synthesis in...
Drug action
Ataluren restores the synthesis of dystrophin by allowing ribosomes to read through premature stop codons that cause incomplete dystrophin synthesis in nonsense mutation Duchenne muscular dystrophy.
Indications and dose
**Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients (initiated by a specialist)**
- **Adult** (By Mouth): (consult product literature).
**Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients (initiated by a specialist) for ataluren**
- **Child 2-17 years (body-weight 12 kg and above)** (By mouth): (consult product literature).
Side effects
Common or very common Appetite decreased; constipation; cough; enuresis; fever; flatulence; gastrointestinal discomfort; haemorrhage; headache; hypertension; hypertriglyceridaemia; nausea; pain; skin reactions; vomiting; weight decreased Frequency not known Ear infection; malaise
Interactions
**Other interactions (21):**
- Aminoglycosides Ataluren should not be co-administered with intravenous aminoglycosides, based on cases of decreased renal function observed in a clinical trial in patients with nmCF (see section...
- Elevations of serum creatinine occurred in several nmCF patients treated with ataluren and intravenous aminoglycosides together with other antibiotics for cystic fibrosis exacerbations.
- The effect of co-administration of ataluren with other nephrotoxic medicinal products is unknown.
- Patients should maintain adequate hydration while taking ataluren (see section 4.4).
- Effect of other medicinal products on ataluren pharmacokinetics Based on in vitro studies, ataluren is a substrate of UGT1A9.
- Co-administration of rifampicin, a strong inducer of metabolic enzymes including UGT1A9, decreased ataluren exposure by 29%.
Pregnancy
Manufacturer advises avoid-toxicity in animal studies.
Breast feeding
Manufacturer advises discontinue breastfeeding-present in milk in animal studies.
Renal impairment
Manufacturer advises close monitoring-safety and efficacy not established.
Medicinal forms
Clinical governance
**Author:** MedNext Clinical Team. **Clinical reviewer:** Dr Shameer Deen, MBBS, MS, MRCS. **Sources:** BNF, Indian Pharmacopoeia, CIMS India. **Correction:** Report errors at support@mednext.academy.
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