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Homology Medicines (NASDAQ:FIXX) has announced that the US Food and Drug Administration (FDA) has given it Fast Track designation for HMI-102, a gene therapy to potentially treat adults with phenylketonuria (PKU). As quoted in the press release: PKU is an inborn error of metabolism primarily caused by mutations in the PAH gene that lead to phenylalanine hydroxylase … ContinuedNEXT 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...
Biological therapy involves the use of living organisms, substances derived from living organisms, or laboratory-produced versions of such substances to treat disease. Some biological therapies for cancer use vaccines or bacteria to stimulate the body&rs...