Chicago, Ill. – 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.
For further details about the conference, including registration and abstract submission, please click here.
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.
Conference details
When: March 3-6, 2024
Venue: Hilton Orlando – 6001 Destination Parkway, Orlando FL 32819