The 62-week results from a phase 2b trial of eteplirsen show the drug continued to stabilize walking ability in boys with Duchenne MD
posted on December 7, 2012 - 4:57pm
Eteplirsen, an exon-skipping drug in development by Sarepta Therapeutics to treat Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin gene, continues to show benefit at 62 weeks with respect to the distance walked in six minutes.
Researchers are studying the 'natural history' of centronuclear/myotubular myopathies, central core disease and multiminicore disease, with an eye to future trials
posted on March 23, 2012 - 12:17pm
Investigators at the University of Michigan in Ann Arbor are conducting a study of the progression of centronuclear myopathies (CNMs), including the type known as myotubular myopathy (MTM); central core disease (CCD) and multiminicore disease. The study is intended to:
A biomarker is any biological indicator that doctors or researchers can objectively measure and evaluate to determine the state of an individual’s health; confirm disease onset and progression; or gauge whether an experimental treatment is working or not.
No matter how you look at it, the losses associated with the progression of ALS are always difficult and hugely significant.
For artists, this loss goes beyond the inability to make a living or participate in a favorite hobby. Painting, writing, singing, playing music and other art forms are artists’ means of expressing themselves, thereby releasing their internal stressors and emotional energy.