Epidermolysis bullosa and global challenges in translational research for rare diseases

Rare diseases collectively affect hundreds of millions of individuals worldwide; however, each condition individually faces substantial barriers to research, innovation, and equitable access to therapies. Accelerating and promoting research in rare diseases is therefore not only a scientific necessity but also a public health and ethical imperative. Limited funding, small patient populations, fragmented expertise, and complex regulatory and reimbursement pathways frequently delay the translation of scientific advances into tangible clinical benefits, particularly outside high-income regions. Even when innovative therapies receive [...]