Fig. 4From: Opportunities for developing therapies for rare genetic diseases: focus on gain-of-function and allosteryDiseases with gain-of-function mutations have higher chance to have treatment under development. Percentages of rare diseases covered by at least one FDA-designated orphan drug were plotted across categories. Mendelian diseases with a gain-of-function mutation and late clinical onset has the highest chance to be readily covered by a orphan drug (16 vs. 8% for all rare diseases)Back to article page