With its recent data drop for an oligonucleotide candidate, Dyne Therapeutics signals it may become a frontrunner in this ...

While some forms of MD, like Duchenne Muscular Dystrophy, primarily affect boys due to dystrophin gene mutations, others, like Limb-Girdle and Myotonic MD, have distinct patterns and impacts.

Other symptoms may include: Often children with Becker muscular dystrophy can walk. As they get older they may need to use a cane or wheelchair to get around. The symptoms of myotonic dystrophy ...

The disease limits their life expectancy to just 20 years old. The most common form in adults is Myotonic muscular dystrophy. Apart from the muscle wasting, it can affect other organs such as the ...

In recent years, a growing understanding of myotonic dystrophy type 1 (DM1 ... making it the most common adult-onset muscular dystrophy. We believe that the market opportunity for a new DM1 ...

Objectives: To determine risk factors for cardiac complications in patients with myotonic dystrophy, and to determine whether permanent cardiac pacemakers may be beneficial in the treatment of ...

The FDA has granted fast-track designation to an investigational antisense oligonucleotide for those with myotonic dystrophy ... as possible,” Doug Kerr, MD, PhD, chief medical officer of ...