cALD

Skysona HCP Global Campaign Launch

Cerebral Adrenoleukodystrophy (cALD) is a devastating, relentlessly progressive neurological disorder and this was our opportunity to show why Skysona should be the treatment of choice for this relentlessly progressive neurological disease. As a breakthrough gene therapy, Skysona can change the standard of care for this often fatal disorder and finally give the community greater hope that patients (young boys) can live full lives.

Although those treating cerebral cALD are top specialists in their field and are familiar with gene therapy, utilizing this type of treatment for patients will be novel. Despite the challenges and risks of current care, treaters have grown accustomed to allo-HSCT transplant procedures over the years. The benefits of Skysona over transplants are clear but often aversion to what’s new can enable a slow uptake. We needed to prove that doctors should be on the foreground of changing what’s possible when treating cerebral ALD and encourage their colleagues to do the same.

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