Translarna

About Translarna

• Translarna, containing Ataluren, is a protein restoration therapy for genetic disorders caused by nonsense mutations, which prematurely halt essential protein synthesis, resulting in non-functional proteins like dystrophin in Duchenne muscular dystrophy.
• Duchenne muscular dystrophy is a fatal genetic illness that primarily affects men. It causes progressive muscle weakness starting in early childhood and results in early death in the middle of the 20s from heart and respiratory failure. 
• Translarna treatment slows the progression of disease and helps to improve the muscle function by expression of full-length protein.

Strengths:

Translarna is available in three strengths; 125mg, 250mg, and 1000mg.

Recommeded Dosage: 

The recommended dosage for Translarna differs from patient-to-patient according to their body weight to ensure that each individual should receive the correct dosage. It should be taken with or without food.

• The medicine should be taken orally three times a day: morning, midday, and evening. 
• There should be 6-hour intervals between morning and midday doses, 6-hour intervals between midday and evening doses, and 12-hour intervals between the evening and the next day’s first dose.
• The recommended dosage is 10mg/kg of body weight in the morning, 10mg/kg at midday, and 20mg/kg in the evening. 
• Mix the medicine into a suspension with at least 30 ml of liquid (water, milk, fruit juice) or 3 tablespoons of semi-solid food. 
• Ensure thorough mixing before administration. Adjust the amount of liquid or semi-solid food to suit patient preference, and the entire dose should be consumed.

Important: 

Patients should always consult their doctors or healthcare professional before taking Translarna. There may be drug interactions, Inform the doctor about all of the drugs you are taking, including prescription, over-the-counter, and herbal supplements. Translarna can affect your ability to drive or operate machinery. Before operating machinery or driving a vehicle while taking Translarna, be sure to talk to your doctor.

Warning & Precautions

• In case of an allergy to Translarna, do not use it without informing your doctor. 
• Only ambulatory DMD patients with a nonsense mutation in the dystrophin gene are permitted to take Translarna. 
• Translarna can interact with other medications. It is important to let your doctor know about all the drugs you are taking, including prescription, over-the-counter, and herbal supplements. 
• While taking Translarna with drugs that stimulate UGT1A9 or that are substrates for OAT1 or OAT3, caution should be used.
• There are no adequate data from the usage of Translarna in pregnant women. Only at dosages that also caused maternal toxicity in animal studies did reproductive damage become apparent. It is not advised to take Translarna when pregnant. 
• While receiving Translarna therapy, breastfeeding is not advised. Translarna excretion in human milk is unknown. Translarna and its metabolites are excreted in milk, according to available pharmacodynamic and toxicological data in animals. 
• Birth defects may be caused by Translarna. Consult your doctor before using Translarna if you are pregnant or intend to become pregnant. 
• Any liver or kidney issues should be mentioned to your doctor. 
• Any allergies you may have, including those to other substances or foods, should be disclosed to your physician. 
• Translarna should not be taken with intravenous aminoglycosides, such as gentamicin and tobramycin. This is because aminoglycosides can reduce the efficacy of Translarna and increase its toxicity.
• Vancomycin and Cisplatin are two drugs that should not be taken with Translarna if the patient have issues in kidneys.
• Alcohol and Translarna can interact, raising the possibility of adverse effects. So avoid consuming alcohol while taking this medicine.. 
• Grapefruit and grapefruit juice have the potential to interact with Translarna and raise the danger of adverse consequences. 

Common Translarna Side Effects 

•     Diarrhoea
•     Nausea
•     Vomiting
•     Headache
•     stomach ache or discomfort
•     Gas (Flatulence)
•     loss of appetite
•     Loss of weight
•     elevated triglycerides
•     Hypertension (High blood pressure)
•     Cough
•     Nose bleeding
•     Abdominal discomfort
•     Constipation
•     Rashes
•     Muscle aches in the arms, legs, and chest
•     Blood in the urine

Use in Specific Population:

The safety and effectiveness of Translarna in patients aged 65 and older have not been established. No dosage adjustment is necessary for patients with mild or moderate renal impairment. The use of Translarna is not recommended for patients with severe renal impairment (eGFR <30 ml/min) or end-stage renal disease. 

No dosage adjustment is needed for patients with mild, moderate, or severe hepatic impairment. Pediatric patients with a body weight of 12kg or more should follow the dosing recommendations based on body weight. 

Storage and Handling:

• This medication does not necessitate any specific storage conditions. It should be taken promptly after preparation. 
• If the prepared dose is not consumed within 24 hours and has been refrigerated (2°C – 8°C), or within 3 hours at room temperature (15°C – 30°C), it should be discarded. 
• Please consult your pharmacist for guidance on the proper disposal of unused medicines, and remember that these actions are essential for environmental protection.