fibrosis

This report on treatment development for Duchenne muscular dystrophy contains news about fighting fibrosis (scarring), treating DMD-related heart disease, exon skipping and the corticosteroid deflazacort

posted on February 2, 2015 - 12:04pm
Development of treatments for Duchenne muscular dystrophy (DMD) continues to advance. Many of the new investigational drugs are potentially applicable to all DMD patients, while a few target those with specific mutations in the dystrophin gene. Fighting fibrosis

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.

Insights from pediatric neurologist Carsten Bönnemann

posted on July 1, 2013 - 9:23am
Quest Vol. 20, No. 3
Update (March 24, 2015): A phase 1 trial of omigapil in children and adolescents 5-16 years old who have merosin-deficient congenital muscular dystrophy and meet other study criteria is open in Bethesda, Md. See Assessment of Safety and Tolerability of Omigapil (CALLISTO), or enter NCT01805024 in the search box at ClinicalTrials.gov.

Drug testing for Duchenne MD, Leigh syndrome and Pompe disease moves forward; and two MDA-supported mouse studies suggest leads for myasthenia gravis and periodic paralysis

posted on March 26, 2013 - 4:21pm
Drug development and identifying new leads for possible drug development are in the news for five neuromuscular diseases in MDA’s program.
posted on March 31, 2011 - 11:34am
QUEST Vol. 18, No. 2
In May 1997, Kathryn Wagner was doing something she hadn’t had much time to do in years: She was watching television. Wagner had just given birth to her first baby, James, and was on leave from her postgraduate training in neurology at Johns Hopkins University School of Medicine in Baltimore.

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.

A change in the LTBP4 protein gene reduces muscle-damaging signals in mice with a disease resembling LGMD2C

posted on November 9, 2009 - 9:46am
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.

Blocking a protein associated with inflammation and scarring helped mice with a DMD-like disease

posted on May 22, 2009 - 4:23pm
A protein called osteopontin has been implicated as a cause of some of the detrimental inflammation and scarring ("fibrosis") of muscle tissue that takes place in Duchenne muscular dystrophy (DMD). Eliminating osteopontin was beneficial to mice with a DMD-like disease, and the researchers concluded that reducing osteopontin should be investigated as a possible therapy for DMD.