Five Questions with Researcher Erik Storkebaum
Erik Storkebaum, of the Max Planck Institute for Molecular Biomedicine in Germany, is studying the underlying mechanisms that drive CMT.
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Five Questions with CMT Researcher Kleopas Kleopa
Kleopas Kleopa, professor at Cyprus School of Molecular Medicine, received an MDA grant to test a treatment in CMT1X
Jerry Lewis’ Work Helped Scientists Discover the DMD Gene
In October 1986 Jerry Lewis announced the DMD gene had been discovered, a monumental moment in MD research.
Remembering Jerry — and a Long Ride to Meet Him
Donnie Foster, who lives with CMT, shares his story of trekking from MO to Vegas to meet Jerry Lewis at the telethon.
There’s Life With CMT, and There’s Also Everyday Life
Lindsey Baker, who lives with CMT, writes about adjusting to life after the end of a relationship.
With One Finger, AJ Brockman Paints the World
AJ Brockman, an artist who lives with SMA, uses only the index finger on his left hand to create works of art.
MDA Awards Human Clinical Trial Grant for Myotonic Dystrophy Natural History Study
MDA has awarded a human clinical trial grant to Nicholas Johnson, M.D., to conduct a natural history study in congenital DM1.
Following Disappointing Phase 3 Results, Ultragenyx Discontinues Ace-ER Program for GNEM/HIBM
A phase 3 study for the treatment of GNE myopathy (GNEM), also known as hereditary inclusion-body myopathy (HIBM) and Nonaka myopathy, failed to meet its primary and key secondary endpoints.
Expanding the ABLE Act
August is ABLE to Save month, and in celebration MDA would like to highlight the achievements of the Achieving a Better Life Experience (ABLE) Act and ways to continue its success.
FDA Reauthorization Act Signed Into Law
MDA thanks Congress and the administration for working together in a bipartisan effort to pass the FDA Reauthorization Act.
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