Exercise Associated with Preservation of Strength in CMT Study
Results from an MDA-supported study show that resistance exercise not only is safe, but can help reduce muscle weakness experienced by people with CMT.
Tag: Research Advances
Five Questions with Researcher Johanna Hamel
Learn more about researcher Johanna Hamel’s clinical research training fellowship.
Becoming a Science Person, Sort Of: My First Research Blog
Joe Akmakjian shares highlights from his interview with MDA grant recipient Mattia Quattrocelli.
FDA Grants Orphan Drug Designation to Octapharma USA’s Experimental Dermatomyositis Drug Octagam
Octapharma USA’s investigational drug IVIG (brand name Octagam) to treat dermatomyositis, has received FDA orphan drug designation.
MDA to Host Emflaza Webinar July 6
DMD Families are invited to an Emflaza webinar in partnership with PTC Therapeutics on July 6.
AMO Pharma’s Myotonic Dystrophy Drug AMO-02 Receives FDA Fast Track Designation
The investigational drug AMO-02 (tideglusib) has received FDA fast track designation for the treatment of DM1.
For the Love of Devin: Josh Argall Races to Find a Cure for His Son — and All MDA Families
This Father’s Day, we’re recognizing an extraordinary MDA dad. Josh Argall was initially devastated by his son Devin’s DMD diagnosis, but once the shock wore off, he resolved to do everything he could for his son and all MDA families. “I can’t discover the new medicine, I can’t provide healthcare, but I can contribute to the mission in my own way, so that’s what I decided to do…There isn’t anything I wouldn’t do for MDA. Knowing how these parents feel and these children feel, that’s what keeps me going.”
Advisory Committee Meeting Scheduled for Translarna
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.
Spinraza Helps Young Woman Participate in Muscle Walk — and She Thinks You Should, Too
Kate, who lives with SMA type 3 and has directly benefited from, Spinraza, a medication that can trace its root back to MDA, encourages supporters to go the distance for MDA via their local MDA Muscle Walks.
Questions and Answers about Newly FDA-Approved Radicava to Treat ALS
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.
