How the FDA tries to balance urgent needs against real risks when evaluating new drugs
Update (April 15, 2014): This story has been updated to reflect that there is now a new FDA office, the Office of Health and Constituent Affairs, whose job it is to serve as a liaison between the FDA and various outside stakeholders, including patient advocates and consumers. See Resources, below.
A drug approved in Europe to treat Lambert-Eaton myasthenic syndrome is now being tested in the United States; clinical trial participants sought
BioMarin Pharmaceutical of Novato, Calif., is conducting a multicenter study of 3,4-diaminopyridine phosphate (3,4-DAP), also known as amifampridine phosphate, in adults with Lambert-Eaton myasthenic syndrome (LEMS).
There are eight U.S. trial sites, with additional sites planned for France, Germany, Italy, Poland and Spain.
"Lumizyme has stopped the progression of my disease," says one of first to receive the drug since the FDA granted commercial approval
Update (Aug. 4, 2014): The U.S. Food and Drug Administration (FDA) has expanded the approval of Lumizyme so that it can now be used to treat Pompe disease patients of all ages, including children younger than age 8. See FDA Expands Approval of Drug to Treat Pompe Disease to Patients of All Ages; Removes Risk Mitigation Strategy Requirements, Aug. 1, 2014.
The Food and Drug Administration has OK'd the first U.S. treatment specifically for late-onset Pompe disease
The U.S. Food and Drug Administration (FDA) has approved U.S. sales of the enzyme-replacement drug Lumizyme, the first treatment in the U.S. specifically for late-onset Pompe disease.
Genzyme seeks FDA ok for Pompe treatment