Five Questions with Researchers Nigel Laing, Kristen Nowak and Rhonda Taylor
Dr. Nigel Laing was awarded an MDA research grant to test two potential treatments for McArdle disease.
Author: Amy Madsen
Simply Stated: What is CMT?
September is #CMTAwareness Month, but do you really know what CMT is and what causes it? Learn more:
CENTAUR ALS Trial Seeks Participants
Researchers are looking for people with ALS to join a clinical trial to test the drug AMX0035.
Golodirsen Shows Potential to Treat DMD
Sarepta announced encouraging results from a phase 1/2 DMD clinical trial for golodirsenm which targets exon 53.
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
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.
