30-person trial will test the safety, tolerability and pharmacokinetics of the experimental drug HT-100 in boys with Duchenne muscular dystrophy
posted on July 30, 2013 - 1:51pm
Update (Jan. 6, 2014): In a Dec. 23, 2013, announcement, Halo said the U.S. Food and Drug Administration (FDA) had placed this trial on "clinical hold" because of some adverse events that occurred in dogs being treated with HT-100. No further dosing of patients will occur until this issue has been resolved.
A change in the LTBP4 protein gene reduces muscle-damaging signals in mice with a disease resembling LGMD2C
posted on November 17, 2009 - 5:00pm
New research has shown that a change in a gene not previously connected to type 2C limb-girdle muscular dystrophy (LGMD2C) modifies the severity of the disease in mice and is likely to do the same in people with this and perhaps with related types of muscular dystrophy.