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Patients with late-stage Pompe disease who are failing to respond to enzyme replacement therapy will now have early access to an alternative treatment option, after Amicus Therapeutics' cipaglucosidase alfa with miglustat was approved for the UK's ...
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has renewed for a further year the Early Access to Medicines Scheme (EAMS) scientific opinion for Santhera's Duchenne muscular dystrophy (DMD) drug idebenone.
UK patients with a form of advanced lung cancer will soon be able to access the immunotherapy drug Tecentriq (atezolizumab) in combination with Avastin (bevacizumab), carboplatin and paclitaxel chemotherapy through.
Akcea Therapeutics’ volanesorsen will be available to eligible patients in the UK before it is officially licensed in the EU, having been granted entry to the country’s Early Access to Medicines Scheme.