Pearson Syndrome

Stanford University is collecting samples of blood, muscle and other tissues from people with neuromuscular disorders for use in research

posted on January 7, 2015 - 12:06pm
Ever wondered how someone with a neuromuscular disorder in his or her family might contribute to research efforts in this field?

The rapid pace of research, unprecedented number of treatments in clinical trials and opportunities to share ideas across diseases made for an exciting 2013 MDA Scientific Conference

posted on April 25, 2013 - 4:51pm
Turning neuromuscular disease research into treatments as quickly and effectively as possible was the overarching theme of dozens of formal presentations, nearly 200 scientific posters, and countless informal conversations at the MDA Scientific Conference, April 21-24. A palpable sense of excitement pervaded the sold-out event thanks to the unprecedented number of experimental treatments in...

EPI-743, an experimental compound that helps mitochondria generate energy, is being tested in mitochondrial and metabolic disorders; a trial in Friedreich's ataxia is planned

posted on November 16, 2012 - 5:00am
Update (Jan. 7, 2014): The trial of EPI-743 in Leigh syndrome is no longer accepting new participants.

Research items about Friedreich's ataxia, myasthenia gravis, mitochondrial myopathies, type 1 myotonic dystrophy, gene therapy and gene modification

posted on July 7, 2011 - 10:28am
Edison drugs target FA, mitochondrial diseases