Until very recently, the only treatment available for DMD was corticosteroids. While clinicians are likely to be well aware of corticosteroids and their utility in the management of DMD, they are less likely to be aware of newer therapeutic options. Given the multisystem involvement of DMD, multidisciplinary management is important, yet this is still not a standard practice. Physicians are likely unaware of mutation-specific targeted therapies presently under development for LGMD. While there are no therapies presently approved for LGMD, several gene therapy trials in clinical stages are underway. These videos share the latest updates in these areas as presented at a recent neuromuscular meeting.
Upon completion of the activity, participants should be able to: