It is estimated that there could be 100,000 patients affected by BCD globally, which means on average, three people become blind due to BCD every day. Currently, there is no approved treatment available, but there is hope. We listed a few potential treatments here, one of which is driven by a BCD patient. Their gene therapy showed promise in treating BCD in preclinical studies. It has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA). However, a lot more effort and resources are needed to advance BCD treatment R&D into human clinical trial. With our combined wisdom and effort, we will make this a reality.
Here we also gathered and summarized BCD related research, which have brought more understanding of this rare disease, improved correct diagnosis and provided insight on potential treatments.