Rare disease families struggle as biotechs back out and NIH adds new clinical trials.

Families of patients with rare diseases have raised millions of dollars to fund research and development of potential therapies, only to see biotech companies back out of the projects. In some cases, the families have had to take on the work themselves, partnering with academic institutions to advance the research. Despite the challenges, some of these efforts have resulted in promising gene therapies and other treatments that are now in clinical trials.