30-person trial will test the safety, tolerability and pharmacokinetics of the experimental drug HT-100 in boys with Duchenne muscular dystrophy
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.
Jim Rusche from Repligen Corp. announced encouraging interim results from a phase 1 clinical trial of RG2833 in Friedreich's ataxia
Interim results from a phase 1 clinical trial of RG2833 in people with Friedreich’s ataxia (FA) show that the experimental drug is well-tolerated, and that it appears to increase the activity of the gene for the frataxin protein.
A phase 1 trial of Ultragenyx’ treatment for hereditary inclusion-body myopathy caused by GNE mutations is taking place in New York and California
Update (Feb. 11, 2013): The phase 2 trial is no longer recruiting participants.
A phase 3 trial of the drug dichlorphenamide in hyperkalemic and hypokalemic periodic paralysis is open at several U.S. and European centers
A multicenter study of the drug dichlorphenamide in 140 adults with hyperkalemic or hypokalemic periodic paralysis is open at sites in California, Kansas, Massachusetts, Minnesota, Missouri, New York, Ohio and Texas, as well as in France (not yet recruiting), Italy and the United Kingdom.
The following article contains items about: Friedreich's ataxia, Charcot-Marie-Tooth disease, myotonic muscular dystrophy type 1, amyotrophic lateral sclerosis, Emery-Dreifuss muscular dystrophy and distal muscular dystrophy (Miyoshi myopathy)