Post by California Institute for Regenerative Medicine (CIRM)

At UCLA Health’s California Center for Rare Diseases, “rare” represents more than 7,000 conditions affecting 3 million Californians. Through multidisciplinary care, genetic diagnosis, registries, clinical trials, and novel therapies, UCLA is advancing rare disease research and treatment in real time. Recent milestones reflect that momentum, from the launch of the nation’s first Multidisciplinary Bloom Syndrome Clinic to advances in gene therapy for Duchenne muscular dystrophy and other genetic conditions. Among these efforts is promising investigational gene therapy research led by Dr. Donald Kohn, whose work, funded by CIRM, is helping expand what may be possible for families affected by rare diseases. Stories like these really highlight how important it is to keep investing in research that translates into real treatments, and how working together in teams can help bring new therapies to patients. Read more: https://bit.ly/4tUh4Z5