by Memberzero ·
SS-31 actually got FDA approved in 2025 by Stealth BioTherapeutics for Barth syndrome. What's interesting is the randomized study only had **12 people** and it wasn't even a perfect study where every endpoint came back positive. The six minute walk test and fatigue scores didn't show a significant improvement during the randomized part but the longer term results ended up looking better. The FDA gave it accelerated approval and now they still have to do a confirmatory study. Pretty interesting seeing SS-31 actually make it through the FDA with data like this especially after being in the research space for so long. I'm curious what that confirmatory study ends up showing.