outcome measures

A study to determine the usual disease course of myotubular myopathy has sites in North America and France

posted on March 18, 2014 - 11:41am
Update (May 20, 2014): This story has been updated to reflect additional information received May 20, 2014, from Michelle Nelken at Valerion Therapeutics, including the joint sponsorship of this study by Valerion and Genethon. ====================================================================================

A study to determine the natural history and outcome measures for clinical trials in Becker MD and sporadic (nongenetic) inclusion-body myositis is open in Ohio

posted on November 18, 2013 - 5:00am
A study to determine the best outcome measures — ways to evaluate the effects of a treatment — for use in clinical studies in Becker muscular dystrophy (BMD) and sporadic (nongenetic) inclusion-body myositis (sIBM) is underway at Nationwide Children's Hospital in Columbus, Ohio, under the supervision of neurologist Jerry Mendell working with physical therapists Linda Lowes and

Differences in motor, cognitive and language function can be assessed in very young boys with Duchenne MD; the finding may help develop outcome measures for DMD clinical trials

posted on June 19, 2013 - 10:51am
Careful assessments of motor, cognitive and language function in very young children with Duchenne muscular dystrophy (DMD) show these children lag behind their age-matched peers well before obvious symptoms of the disease appear.