Commentary
Video
Preparing for the Future of Rare Disease Treatment: Robert Califf, MD
Author(s):
The former FDA commissioner discussed the collaborative power of the neuromuscular community and the challenges of implementing emerging therapies in a rapidly evolving care landscape. [WATCH TIME: 4 minutes]
WATCH TIME: 4 minutes
"We don’t solve problems as individuals in areas like this—it takes teamwork between scientists, clinicians, patients, and families to make real progress."
In recent years, neuromuscular diseases have become a growing focus of investment and innovation, fueled by advances in genetic science, novel therapeutic platforms, and increased public and private funding. Conditions like Duchenne muscular dystrophy, spinal muscular atrophy, amyotrophic lateral sclerosis, and facioscapulohumeral muscular dystrophy–once considered untreatable–are not the focus of robust clinical pipelines, with investigational treatments targeting the underlying molecular and genetic causes.
Because of the similarities and overlap in symptoms with several of the prominent neuromuscular disorders, there has been expressed interest from clinicians to collaborate more consistently with other disease states and areas of expertise. The 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific conference, held March 16-19, in Dallas, Texas, was a perfect example of this, bridging numerous clinical focuses and subspecialties in one central location for the latest in research and care updates. The meeting was kicked off by former FDA commissioner and cardiologist Robert Califf, MD, who delivered a keynote speech on the advances in neuromuscular medicine and the need for continued funding efforts.
Following his presentation, Califf sat down with NeurologyLive® to reflect on the power of collaboration across the neuromuscular community–from scientists and clinicians to families and patients. In the discussion, he also talked about the necessary steps the clinical community will need to take as more novel, innovative therapeutics emerge. Califf, who founded and directed the Duke Clinical Research Institute, warned of major structural challenges, including the shortage of trained neurologists, the need for post-marketing surveillance systems, and the importance of sustainable access models.
