While currently there are no disease-modifying therapies approved to treat XLMTM, several novel therapeutic strategies have shown promise in pre-clinical disease models, including tamoxifen [20], PIK3C2B inhibition [21], dynamin 2 reduction [22], and intramuscular injections of either adeno-associated virus (AAV)-shRNA or AAV-mediated gene replacement therapy (resamirigene bilparvovec) [23–26]. Here, PIK3C2B is linked to X-linked myotubular myopathy.