Challenge A rare disease patient advocacy group based in the US was receiving requests from patients in outlier countries who wanted to gain access to an EMA- and FDA-approved product not commercially available in [...]

Challenge A patient advocacy group supporting patients with a rare metabolic disorder in the western Pacific wanted to help provide access to effective treatments. There are currently no approved medicines available for this condition and, [...]

Challenge A patient living in the Caribbean had been suffering from a genetic lung disorder since a young age and had found there were no effective treatments available in his home country. His local physician [...]

Challenge A patient in the US suffering from a seriously debilitating disease was struggling to manage his condition. The patient had tried all available treatment options in the US, but none had been effective. The [...]