Astellas therapy brings new hope to rare disease patients amid broader biotech challenges

A new Astellas treatment is being highlighted as a source of optimism for a community affected by a rare disease, after a period of setbacks in the program.

The article also notes a revived gene therapy effort targeting the same condition, while reporting that recent Alzheimer’s drug uptake has been lower than expected.

Separately, the piece points to ongoing uncertainty at the FDA, where questions are being raised about the future of Commissioner Marty Makary.