Gene therapy may cure rare diseases. But drug dealers have few incentives, leaving families desperate
by Robin Alderman is faced with a painful reality: gene therapy could cure her son Camden’s immune deficiency. But it is not available to him. In …
Tag: Wiskott-Aldrich syndrome
Takeaways from AP report on access to gene therapies for rare diseases
The promise of gene therapy is huge for families dealing with rare genetic disorders. Such treatments offer the possibility of one-time cures. But families and …