It is hoped that the approach developed in this study will be rapidly incorporated in standard clinical practice to maximise the diagnosis of rare genetic disorders. (sciencedaily.com)
But will approving the drug pave the way for all manner of pharmaceuticals to treat rare genetic disorders? (newscientist.com)
Its teams are launching clinical trials in rare genetic disorders affecting the eye and the skin aiming at getting treatments from 2017. (istem.eu)