Clinical Studies

FDA approval for a rare disease treatment is not a finish line. It is one important milestone in a broader commitment to working with a rare disease community to meet their needs respectfully and responsively. We remain focused on generating additional data to support the safe and effective use of therapies beyond the evidence that supported FDA approval. To that end, we currently have an observational study underway to better understand the real-world use of givinostat.

The goal of this study is to learn more about how the product affects individuals who are taking the medication as part of their routine care. All assessments are performed at the time of a routine clinical care visit according to clinical practice, and data will be extracted from the medical records.