This phase II clinical trial investigated whether a drug called tideglusib could be a potential treatment for congenital and childhood-onset myotonic dystrophy. Most participants who received the drug for 14 weeks had improved cognitive function and felt less fatigued. The drug was also well-tolerated and safe.
8 (35%). Biventricular hypertrophy and four chamber dilation were found in 3 (25%) of 12 cases in a study by Nguyen et al.,
Current research is focusing on how we might be able to one day use gene-editing technology or other treatments to remove the clumps of RNA that cause the symptoms of myotonic dystrophy. However, this therapy is not yet possible in humans.
8 (35%). Biventricular hypertrophy and four chamber dilation were found in 3 (25%) of 12 cases in a study by Nguyen et al.,
Current research is focusing on how we might be able to one day use gene-editing technology or other treatments to remove the clumps of RNA that cause the symptoms of myotonic dystrophy. However, this therapy is not yet possible in humans.