Our March Myology Café took place on Thursday 28th March on Zoom. Our expert speaker was Matthew Alexander, from the University of Alabama at Birmingham, USA. Dr Alexander was hosted by Hernan Gonorazky from our Myology Developments Across the World Committee and who works at SickKids in Toronto, Canada.

Dr Alexander joined the meeting to share his experiences of The Muscular Dystrophy Association (MDA) 2024 Clinical and Scientific Conference, which took place in Orlando, Florida (USA) earlier in March, with more than 1,500 attendees. The annual MDA Conference brings together basic and translational scientists, clinicians and industry alongside patients and patient advocacy organisations to better understand and find new treatments for muscle disorders. 

Dr Alexander highlighted several key talks and panel discussions from the conference. The opening address by MDA CEO Donald Wood described the organisation’s progress in bringing together research and advocacy since its foundation in 1950. Next year (2025) will mark the MDA’s 75th anniversary.

The keynote address was given by Brooke Eby (@limpbroozkit on social media), a young woman living with ALS who has gained a large social media following due to her candid discussions online about what her daily life is like. She gave an excellent talk and highlighted the speed at which patients like her need new treatments for muscle disorders. The MDA Legacy Award was presented to Dr Jeffrey Chamberlain for his work on adeno-associated viral (AAV) vectors and gene therapies. There was an excellent panel discussion led by MDA Chief Research Officer Sharon Hesterlee, PhD on neuromuscular disorders in the age of gene therapies.

Some key speaker highlights, in particular the clinical trial updates, were Dr Jerry Mendell’s presentation of the delandistrogene moxeparvovec (Elevidys) Versus Placebo (EMBARK) Sarepta Therapeutics trial. This talk was standing room only, and Dr Mandell highlighted the need for additional Duchenne muscular dystrophy (DMD) outcome metrics besides the North Star Ambulatory Assessment (NSAA). In the Myology Café, Dr Alexander and Dr Gonorazky discussed the interest and need for multiple outcome metrics for DMD and other neuromuscular disorders and the need to evaluate multiple endpoints for clinical trial evaluations.

Other talks (Dr Chris Heier) discussed the molecular mechanisms for vamorolone (AGAMREE) in mdx and bmx mouse models of Duchenne and Becker muscular dystrophy (BMD). Data was presented on the most recent US Food and Drug Administration (FDA) approved drug givinostat (DUVYZAT) for DMD, which is an HDAC inhibitor compound shown to increase muscle mass and delay muscle weakness symptoms in ambulatory DMD boys. Dr Alexander and Dr Gonorazky discussed key pre-clinical/basic science talks including those presented by Drs Eric Wang, Russell Rogers, Felipe Lopez, Lori Walrath, and Jim Ervasti among others. The 2025 MDA Clinical and Scientific Conference will be held March 16-19 in Dallas, TX.