Gene therapy for CCALD is not available to treat children in Europe due to dispute

Skysona is BlueBird Bio’s gene therapy treatment for childhood cerebral Adrenoleukodystrophy (CCALD). This article discusses the decision Bluebird Bio made to leave the EU after a dispute with Germany, meaning Skysona is not available to treat children in Europe – as a result, children with CCALD have been left with no treatment

 

To read: https://english.elpais.com/science-tech/2023-11-26/skysona-the-gene-therapy-that-saved-darius-life-cannot-help-any-more-european-children.html

 

 

 

 

 

 

 

 

 

Alex - The Leukodystrophy Charity