Sarepta Therapeutics continues to report encouraging data from its extension study of eteplirsen, designed to treat Duchenne MD due to specific genetic mutations
Walking ability and respiratory function in boys with Duchenne muscular dystrophy (DMD) show continued benefit from eteplirsen at 144 weeks (almost three years), the drug's developer announced today. In addition, the intravenously infused drug was well tolerated, with no serious treatment-related adverse events seen.
Questions and answers about the FDA and its process for approving drugs
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.
Four professionals recently talked with Margaret Wahl, MDA’s medical and science editor, sharing their thoughts about access to drugs that have not gone through the full approval process
Can We Have It Now?
Drug development consultant Abby Bronson says early access to a new drug can jeopardize its full approval and long-term availability
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...
Bioethicist Arthur Caplan says early access to drugs should not be determined by drug companies but by independent advisory boards
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...
FDA staffer Richard Klein says people often underestimate the risks and overestimate the benefits of taking medications that have not been through the full FDA approval process
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?
Neuromuscular disease specialist and biotechnology company employee Timothy Miller says it can be a significant risk and financial hardship for a small company to supply a drug that has not been through the full approval process
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...