Takeaways from AP’s report on access to gene therapies for rare diseases

By LAURA UNGAR
AP Science Writer

The promise of gene therapy looms large for families dealing with rare, genetic disorders. Such treatments offer the possibility of one-time cures. But families and researchers worry such therapies will remain out of reach. Collectively, about 350 million people worldwide suffer from rare diseases, most of which are genetic. But each of the 7,000 individual disorders affects perhaps a few in a million people or less. So there’s little commercial incentive to develop or bring to market these one-time therapies to fix faulty genes or replace them with healthy ones. Families are frustrated, and some