Today at the World Muscle Society conference, Audentes Therapeutics announced new data from its ASPIRO study testing a gene therapy for X-linked myotubular myopathy (X-MTM). Eight children have been enrolled to date; six have received a low dose of the gene therapy, one received a higher dose and one has not yet been treated (to act as a control). All treated children continue to show improvements in respiratory and neuromuscular function and no new safety issues have been reported since the company’s update in May 2018. Three children no longer require any ventilation support, which is “an unprecedented outcome in children with a congenital myopathy who have been ventilated from birth”.
Audentes also announced that it plans to hold initial discussions with the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) beginning in the fourth quarter of 2018.
For more information, please see Audentes’ press release.