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In this episode, "Advancing Neuromuscular Care and Research," former FDA commissioner Robert Califf, MD, explored the urgent need for sustained NIH funding to advance basic and translational research, including gene editing, assistive devices, and the integration of artificial intelligence. He emphasized the collaborative spirit of the neuromuscular community, where shared technologies and insights can address multiple disorders. The conversation, which took place at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific conference, highlighted critical challenges, such as clinician shortages, healthcare system strain, and the need for post-market learning to manage uncertainties with emerging therapies like gene therapy. Additionally, he called for innovative funding solutions to address the high costs of treatment, ensure equitable access, and maintain progress in neuromuscular care while fostering long-term health equity.

Episode Breakdown:

1:05 – Funding priorities for neuromuscular research and NIH support

1:55 – Role of gene editing, assistive devices, and AI in care

4:50 – Collaboration and togetherness of the neuromuscular community

6:35 – Considerations as new therapeutics emerge for neuromuscular disorders

8:05 – Funding solutions for equitable care

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