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.
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.
A delegation of dozens of MDA advocates from 23 states visited more than 100 congressional offices on Capitol Hill earlier this week to speak to their representatives about the importance of programs and policies that help accelerate the development of treatments and cures for individuals and families living with muscular dystrophy, ALS and related neuromuscular diseases.
Being chosen to speak in front of the FDA and represent the SMA community makes me feel incredibly honored. Today’s SMA Patient-Focused Drug Development (PFDD) meeting with the FDA means we have a voice as patients living with rare muscle diseases. The PFDD meeting was set up so that patients with SMA and the parents of children with SMA could stand before the FDA and voice their concerns, discuss their experiences with research and address the need for continued research that evolves to include more patients. This is my story.
National Ambassador Joe Akmakjian recently attended the 2017 MDA Scientific Conference. He writes, “I found the conference to be very hopeful and exciting experience. It was an honor to meet the people who have made a career out of saving my life and the lives of the other individuals and families. Everyone connected to MDA should be excited to know that these researchers are actively sharing ideas and discussing ways to solve problems and find the solutions we’re all looking for.”
Over 500 of the sharpest minds in research have gathered at MDA’s Scientific Conference as they work to unlock the secrets of neuromuscular diseases.
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.
New research, supported by MDA and the CMT Association, examines gene therapy in CMT.
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.
PTC Therapeutics announced that the FDA will review its investigational drug ataluren (brand name Translarna) for the treatment of some forms of DMD.
