Summary
Bardet–Biedl syndrome (BBS) is a rare, genetically heterogeneous disorder that affects more than 5,000 individuals in the United States.
A major therapeutic milestone was the approval of IMCIVREE (setmelanotide), the first targeted MC4R agonist therapy addressing BBS-related obesity.
Gene therapy with AXV-101, developed by Axovia Therapeutics, is emerging as a novel option for BBS1-related retinal degeneration.
Bardet–Biedl syndrome (BBS) is a rare, genetically heterogeneous disorder that affects more than 5,000 individuals in the United States. It occurs with equal frequency in males and females and has an estimated prevalence of approxima...
