myotonic dystrophy

Scripps Florida Scientists Create New Approach to Destroy Disease-Associated RNAs in Cells

Scientists from the Florida campus of The Scripps Research Institute (TSRI) have developed a new approach to alter the function of RNA in living cells by…

An important breakthrough in the fight against muscular dystrophies

An important breakthrough could help in the fight against myotonic dystrophy. The discovery, recently published in the prestigious scientific journal Cell,…

In muscular dystrophy, what matters to patients and doctors can differ

However, a new study out this month in the journal Neurology reveals that there is often a disconnect between the two groups over which symptoms are more…

Scripps Research Scientists Create Potent Molecules Aimed at Treating Muscular Dystrophy

While RNA is an appealing drug target, small molecules that can actually affect its function have rarely been found. But now scientists from the Florida campus…

Biologists Find Another Clue to the Origin of Neurodegenerative Diseases

In research published in the March 17 online edition of the journal PLoS Genetics, Tufts University biologist Catherine Freudenreich, and then-graduate student…

Heart Drug Effective for Treating Symptom of Muscular Dystrophy

The findings about the medication mexiletine – a chemical cousin of lidocaine – were published May 4 in the journal Neurology, a publication of the American…

Small molecules might block mutant protein production in Huntington's disease

These man-made molecules also were effective against the abnormal protein that causes Machado-Joseph disease, a neurological condition similar to Huntington’s.The work has been done only in cultured cells, and it will take years before the effectiveness of this process can be tested in patients, the researchers cautioned….