Announcing the 2024 MDA Clinical & Scientific Conference Agenda

The 2024 MDA Clinical & Scientific Conference is just around the corner. We’re looking forward to you joining us, as we showcase groundbreaking research and clinical accomplishments by dedicated professionals working towards preserving and restoring movement in individuals with neuromuscular disease (NMD). With over 50 sessions, 180 speakers, 56 exhibitors and patient advocacy organizations, and 16 Industry Forums, this year’s conference is sure to be as jam-packed with amazing content as always.  

Conference Agenda: 

Conference programming boasts six tracks, focusing on:

• Clinical Considerations 
• Lab to Life 
• Clinical Approaches to Streamlining Care 
• Practical Consideration in Gene Therapy 
• Disease Mechanism & Therapeutic Strategies 
• New, Novel, and Noteworthy: NMD Highlights 

Conference Session Spotlights: 

Practical Considerations in Gene Therapy 

Richard Finkel, MD 

Dr. Finkel is the Director of the Center for Experimental Neurotherapeutics and Endowed Chair in Neurotherapeutics at St. Jude Children’s Research Hospital and .is the distinguished chair of the Practical Consideration in Gene Therapy track 

As part of our “Clinical Considerations” track, this session focuses on the complexities of navigating care decisions for the subset of NMDs with available therapies. Topics will include the relative risks and burdens associated with different types of therapies, the true clinical impact of approved therapies, real-world challenges in equity, and decisions regarding monotherapy vs combination/ sequential therapy. Each topic includes a 15-min panel discussion with audience Q&A. 

Translational Research in ALS: Novel Biomarker and Model Systems 

Rita Sattler, MSc, MD 

Dr. Sattler, of the Barrow Neurological Institute, will host an exciting session, Translational Research in ALS: Novel Biomarkers and Model Systems.  

This session will provide an overview on the most recent discoveries and utilization of molecular bio markers for ALS, which are proposed to be applicable for early diagnosis, but also as a pharmacodynamic marker during clinical trials. In addition, presenters will discuss novel disease models for ALS with the goal of optimizing pre-clinical translational research. 

Where Are We in Gene Therapy in Clinical Practice 

Holly Peay, PhD 

Holly Peay is a Senior Research Public Health Analyst at the Research Triangle Institute (RTI) with extensive experience in the Duchenne muscular dystrophy community, and will host the session, Where Are We in Gene Therapy in Clinical Practice. 

This session will kick off the Practical Considerations in Gene Therapy track, a continuation from 2023. This session is an opportunity for attendees to review the status of Gene Therapy in NMD and gain a deeper understanding of ethical considerations, ongoing and anticipated challenges, misconceptions, and more for clinical practice, as this new approach continues to evolve in NMD. 

The full agenda can be viewed here. 

Conference registration: 

The conference will be held March 3-6 at the Hilton Orlando in Orlando, Florida. Detailed information about the conference and registration can be found here. If you are a patient or caregiver interested in attending the in-person conference, or are an MDA volunteer interested in attending virtually, please fill out this form before registering. 

With your registration, you’ll also gain exclusive access to our on-demand conference content through our virtual event platform after the conference concludes. This content will be accessible for 12 months post-event.  

Members of the NMD community who are registered with MDA are welcome to participate in the virtual conference at no-cost or may register here to attend in-person at the patient/caregiver rate until allotted spaces are filled. Please keep in mind that the level of information presented at this conference will be technical, as this is a professional conference not geared for the general public. For those who would prefer scientific presentations geared for a more general audience, please see current listings on MDA’s Community Education page.