Active substance
Results for the substance ataluren
3 results
About this active substance
Ataluren is a medication used for the treatment of Duchenne muscular dystrophy (DMD) caused by nonsense mutations in the dystrophin gene. It works by facilitating the read-through of nonsense codons during protein synthesis, allowing the production of functional dystrophin protein. Ataluren is particularly indicated for patients who are not eligible for other genetic therapies.
The medication is administered orally, usually three times a day, with the dosage adjusted based on the patient's weight. Treatment is long-term and requires regular monitoring to assess efficacy and safety.
Common side effects include nausea, vomiting, diarrhea, headaches, and upper respiratory tract infections. In rare cases, severe adverse reactions such as renal or hepatic impairment may occur.
Patients receiving ataluren should undergo regular monitoring for renal and liver function, as well as disease progression. Ataluren represents an innovative therapeutic option for patients with DMD, offering a chance to slow disease progression and improve quality of life.
The medication is administered orally, usually three times a day, with the dosage adjusted based on the patient's weight. Treatment is long-term and requires regular monitoring to assess efficacy and safety.
Common side effects include nausea, vomiting, diarrhea, headaches, and upper respiratory tract infections. In rare cases, severe adverse reactions such as renal or hepatic impairment may occur.
Patients receiving ataluren should undergo regular monitoring for renal and liver function, as well as disease progression. Ataluren represents an innovative therapeutic option for patients with DMD, offering a chance to slow disease progression and improve quality of life.
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TRANSLARNA 250mg granules for oral suspension PTC THERAPEUTICS INT ORIGINAL MEDICINE
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TRANSLARNA 125mg granules for oral suspension PTC THERAPEUTICS INT ORIGINAL MEDICINE