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The FDA has given orphan drug designation to a gene therapy product intended for treatment of X-linked retinitis pigmentosa, Applied Genetic Technologies Corp. announced in a press release.The drug candidate, which is intended specifically to treat X-linked retinitis pigmentosa caused by mutations in the RPGR gene, was granted orphan medicinal product designation for the same indication by the European Commission in June 2016, according to the release.
Original Article: FDA gives orphan drug designation for retinitis pigmentosa gene therapyNEXT 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...
Pharmacy is the science and technique of preparing as well as dispensing drugs and medicines. It is a health profession that links health sciences with chemical sciences and aims to ensure the safe and effective use of pharmaceutical drugs. The scope of...