Empowered Patient Podcast

Sheila Frame is the President for the Americas at Amryt Pharma, founded on the belief that rare and ultra-rare diseases were an opportunity to support underserved patients differently than the big pharmaceutical companies could. With the acquisition of Mycapssa, Amryt is changing the treatment for the rare disease acromegaly from painful injections to the first and only twice-daily oral medication. Shelia explains, "We're currently commercially, at least, in three different areas. So lipodystrophy is a pretty significant leptin deficiency for people who suffer from that. It's a fairly rare disease, with about 300 patients roughly in the US. The second one is in homozygous familial hypercholesterolemia, and again, a very rare genetic disease, probably even fewer patients than what we see in lipodystrophy. And then the third area, of course, is acromegaly with Mycapssa, where we actually have products in the market." "I think what's interesting about not only the technology that we have with Mycapssa but also t