Translarna, a new drug under development that may slow functional decline in individuals with DMD cause by nonsense mutations, will be reviewed by an Advisory Committee of the U.S. Food and Drug Administration on Sept. 28, 2017. An FDA decision on the drug is expected on or before Oct. 24, 2017.
Find answers to your questions about access, cost and availability of DMD drug Emflaza.
The U.S. Food and Drug Administration (FDA) on May 5, 2017, approved edaravone (brand name Radicava) for the treatment of ALS (amyotrophic lateral sclerosis). Radicava is the first ALS treatment to be approved in the United States in more than 20 years, and is only the second drug ever approved to treat this devastating disease.
Today, on the first Friday of ALS Awareness Month, the U.S. Food and Drug Administration (FDA) approved edaravone (brand name Radicava), to treat ALS (amyotrophic lateral sclerosis). Under development by Mitsubishi Tanabe Pharma America, Radicava was approved in 2015 to treat ALS in Japan. It’s the first drug to be granted FDA approval to treat ALS in the United States in more than 20 years.
Today the House passed the AHCA, which contains provisions that will impact the health care coverage of individuals and families that MDA serves.
Despite the progress in the quest for treatments and cures, individuals living with the life-threatening diseases that newly approved drugs treat are facing a new challenge — gaining access to the approved therapies. Today, some individuals and families are unable to access FDA approved therapies, which can significantly impact the health and life of the person denied access. Not having access to appropriate therapies is unacceptable. A treatment cannot change the lives of families and individuals if they do not have access to it.
Seven lucky Phoenix-area kids living with neuromuscular diseases received custom adapted bicycles at the recent annual meeting of the American College of Medical Genetics and Genomics.
Excited to try MDA’s new web-based Clinical Trials Finder Tool powered by Antidote? Here are some questions we have received and answers that should make using the tool as seamless as possible.
This week, hundreds of the best and brightest scientists from around the country are gathering in the Washington, D.C., area at the 2017 MDA Scientific Conference, with the goal of working collectively toward advancing scientific breakthroughs to save and improve lives for MDA families.
PTC Therapeutics reached an agreement with Marathon Pharmaceuticals to acquire all rights to Emflaza™ (deflazacort), the first treatment approved in the U.S. for all Duchenne muscular dystrophy (DMD) patients five years and older, regardless of their genetic mutation.
