Golodirsen Shows Potential to Treat DMD
Sarepta announced encouraging results from a phase 1/2 DMD clinical trial for golodirsenm which targets exon 53.
Category: Research
Five Questions with DMD Researcher Mattia Quattrocelli
Dr. Mattia Quattrocelli of Northwestern University was awarded an MDA grant to study the effects of glucocorticoids in DMD.
Five Questions with DMD Researcher Tejvir Khurana
Tejvir Khurana, a professor at the University of Pennsylvania was awarded an MDA grant to test a therapeutic strategy for DMD.
Five Questions with DMD Researcher James Novak
Postdoctoral associate James Novak was awarded an MDA grant totaling $180,000 to study exon skipping drugs in DMD.
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.
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.
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.
Researcher with LGMD Aims to Bring Genetic Diagnoses to People Around the World
Monkol Lek, a researcher at Massachusetts General Hospital and the Broad Institute, both lives with and studies limb girdle muscular dystrophy. He received a research grant from MDA to improve the diagnosis of rare muscle diseases, and this fall he will open his own lab at Yale.
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.
