Genzyme has published an update on the availability of ataluren for Duchenne muscular dystrophy. Ataluren has been tested in clinical trial to treat Duchenne caused by a particular type of muation called a nonsense mutation that affects about 10-15% of boys. You can read about it here:
Ataluren have circulated another update reassuring families that they are making progress with the authorities in Europe to try to allow participants in the clinical trial access to the drug.
The latest update from Genzyme announces real progress – a clinical trial is being planned to further test the drug “ataluren” in Duchenne muscular dystrophy patients who previously participated in the clinical trials in the UK, Europe, Israel and Australia.
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