A new bi-transgenic mouse with fluorescent reporters incorporated into a mini-gene allows sensitive, in vitro monitoring of DM1-related alternative splicing.
Loraine Dressler from Huntington Beach, California is the main caregiver for her grandson Zen, who was diagnosed with congenital myotonic dystrophy at birth. Zen’s severe disabilities haven't stopped her from traveling with Zen, in fact, she has been traveling with him since he was 2 years old. Zen is now 6 years old and Loraine has learned many tips and tricks along the way.
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.