Whether you are newly diagnosed or have been living with a neuromuscular disease for years, there is arguably one guiding principle that is most important when it comes to making decisions about your care and quality of life: You are in charge.
And with good reason.
To people faced with life-threatening diseases, the U.S. Food and Drug Administration (FDA) can seem like an indifferent obstacle, keeping them from treatments that would otherwise be available. But the reality is much more layered and complex.
Each form of muscular dystrophy is devastating in its own way. For some patients, symptoms may manifest at birth or at a young age. My situation was different.
Abby Bronson is the Duchenne muscular dystrophy program manager at Children's National Medical Center in Washington, D.C., where she works with various stakeholders to further therapeutic development for this disease. Bronson has a master's degree in business administration from the University of Pennsylvania and has managed new product development and marketing at biotechnology and...
Arthur Caplan is the William F. and Virginia Connolly Mitty Professor and founding head of the Division of Bioethics at New York University Langone Medical Center in New York City. He holds a doctoral degree in the history and philosophy of science from Columbia University, has authored or edited 32 books and more than 600 papers, and has served on national and international committees as an...
Richard Klein is the director of the Patient Liaison Program at the Office of Health and Constituent Affairs at the U.S. Food and Drug Administration (FDA).
Q: What is the most common misconception that patient communities have about the FDA's accelerated approval mechanism?
Timothy Miller has been a university-associated neurologist specializing in neuromuscular disorders who has recently moved to the biotechnology industry. He has been the director of the MDA-supported pediatric neuromuscular disorders clinic at Children's Clinics for Rehabilitative Services in Tucson, Ariz.; an assistant professor of neurology, pathology and pediatrics at the University of Arizona...
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