Myotonic dystrophy type 1 (DM1) is the most common form of muscular dystrophy.There is little phenotype and genetic data for Chinese DM1 patients. The data to be collected is intended to fill this gap and provide complementary data
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Triplet-primed PCR or Long-read sequencing
Timeframe: Baseline
Muscle Impairment Rating Scale (MIRS)
Timeframe: Baseline through study completion (an average of 1 year)
The modified Medical Research Council (MRC) scale
Timeframe: Baseline through study completion (an average of 1 year)