FDA Issues Guidance on Developing Rare Disease Treatments

FDA Issues Guidance on Developing Rare Disease Treatments

284609

FDA Issues Guidance on Developing Rare Disease Treatments

New guidelines from the U.S. Food and Drug Administration (FDA) may make it easier to develop therapies to treat extremely rare diseases, such as Batten disease. “The release of this guidance from the FDA is a first step and a very welcome one. It speaks to the promise of precise, genetically targeted medicines for rare disease patients with currently unmet needs, and acknowledges the need for thoughtful frameworks to deliver on that promise,” Timothy Yu,…

You must be logged in to read/download the full post.