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NICE has announced that it has decided to approve Alexion Pharma’s Strensiq (asfotase alfa) for the treatment of ultra-rare disease paediatric-onset hypophosphatasia, an inherited condition which affects as few as between one and seven babies in England each year, meaning it can now be accessed on the NHS.
Original Article: NICE agrees approval deal for ultra-rare juvenile bone diseaseNEXT ARTICLE
In order to become availible to pateints, drugs need to undergo a number of phases of clinical trials to test their efficacy and safty and to then be authorised by the drug approval organistion in each respective country. This is the FDA in the USA and N...