A new review article addresses advances in exploiting the biology of RNA with small molecule drug strategies.
A novel genome editing strategy removes expanded CUG repeats in DMPK transcripts.
TREAT-NMD’s TACT process provides unbiased feedback on drug development programs in neuromuscular diseases.
When Suzanne Perkins’ 16-year-old daughter Eliza had trouble opening jars and closing car doors, she took the teenager to the doctor, confident she would pin the problem on too much texting. But when her doctor sent her to a neurologist, Eliza’s diagnosis came quickly and with a wallop.
I am a caregiver. My husband, two of my adult children, and my 6 year old grandson all have DM1. Though I’ve been their wife, mother, and grandmother for many years, it’s only recently that I’ve realized how important the title of my role is … caregiver.
MDF advocate Glen Wiggans, MD recently participated in the evaluation of research applications submitted to the Peer-reviewed Medical Research Program (PRMRP) sponsored by the Department of Defense. Glen was nominated for participation in the program by MDF. As a consumer reviewer, he was a full voting member of the review panel, along with prominent scientists, to help determine how the $330 million appropriated by Congress for Fiscal Year 2018 will be spent on PRMRP research.
A tropical paradise was the ideal spot for the 2018 Myotonic Dystrophy Foundation’s (MDF) Annual Gala, November 8th – 10th. MDF supporters from Hawaii and the mainland came together for three days of fun activities, including: casual and competitive bike excursions, a cocktail party on
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