News

Ask Bio is planning on starting their gene therapy trial for LGMD2i in winter, 2021!  Here is a copy of their latest newsletter: AskBio_AAvenger_Newsletter_Summer 2020
A natural history study in LGMD2i, “Biomarker Development in LGMD 2i”, is now open to enrollment. This study will be collecting data for an upcoming oral treatment study with ML Bio Solutions. For more information on the study and how to participate, please contact Brittney Holmberg at Brittney.Holmberg@vcuhealth.org or 804-552-0014.  Here is the latest press release.
PTC Therapeutics has decided to discontinue A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I). This study was designed to evaluate the safety and efficacy of deflazacort in participants with LGMD2I. Patients currently enrolled in the Emflaza® study that would like to remain on drug are encouraged to work with their physician to pursue access to treatment for up to 6 months.