Posted by Michael Wonder on 23 May 2021
First gene therapy to treat children with rare inherited neurological disease
21 May 2021 - The EMA has recommended granting a marketing authorisation in the European Union for the gene therapy Skysona (elivaldogene autotemcel) for the treatment of children with cerebral adrenoleukodystrophy, a severe form of a rare inherited neurological disease.
This disease, seen almost exclusively in males, affects the brain and leads to an irreversible loss of neurological functions.
