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.