Five years after disaster, a rare disease community gets new chance at treatment Astellas renews trials of gene therapy for XLMTM, hoping for success stories like JJ’s

https://www.statnews.com/2026/05/11/astellas-gene-therapy-trials-resume-promising-myotubular-myopathy-treatment/?utm_campaign=the_readout&utm_medium=email&_hsenc=p2ANqtz-8HruUIBEo9mbFUnc6VaSeW-IQqI6sw_odRZhzXKpU_kjFdmXRMQZycVwNQD5Thpp93tjC8SLX0XfgPqDmRZxk-3rz20A&_hsmi=418165077&utm_content=418165077&utm_source=hs_email By Jason MastMay 11, 2026 Astellas treatment offers new hope to a devastated rare disease community Five years after a gene therapy trial for the fatal muscle disorder XLMTM unraveled amid a string of devastating liver failure deaths, patients and their families are getting a second chance. Astellas has launched a new clinical trial using a next-gen virus engineered to target muscle more precisely at dramatically lower doses, STAT’s Jason Mast writes. The trial is raising hopes for a safer version of a therapy that once helped some children come off ventilators, walk, communicate, and survive against impossible odds. Take, for instance, the astonishing early recoveries of boys like Joshua Jacob Gonzalez — but also the field-shaking deaths that chilled the gene therapy sector. “When the full results of the initial clinical trials are revealed, I believe the world will recognize this work for what it truly is — nothing short of miraculous,” one mother of a son who died of the disease, but who helped fund early research, told STAT. “And still, I hold a measure of trepidation. Concerns remain around liver safety and whether current dosing strategies are sufficient to fully realize the therapy’s potential.”