EFEMP1 and respiratory distress syndrome in premature infants: Accordingly, small molecules or genetic perturbations that alter key cellular responses involved in ML/DHRD progression (e.g., EFEMP1 protein production/turnover, or stress responses due to malformed EFEMP1) have the promise of serving as first-in-human therapies for this rare disease which may comprise up to 0.9% of all inherited retinal dystrophies (IRDs) in select populations [108], amounting to thousands of individuals worldwide.