FDA approves drug to treat Duchenne muscular dystrophy

FDA: The U.S. Food and Drug Administration today approved Emflaza (deflazacort) tablets and oral suspension to treat patients age 5 years and older with Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes progressive muscle deterioration and weakness. Emflaza is a corticosteroid that works by decreasing inflammation and reducing the activity of the immune system. Corticosteroids are commonly used to treat DMD across the world. This is the first FDA approval of any corticosteroid to treat DMD and the first approval of deflazacort for any use in the United States.

A vitamin could help treat Duchenne muscular dystrophy

Lausanne: Researchers are working on a new strategy to combat one of the most severe forms of muscular dystrophy. Rather than acting on the defective gene, they are using large doses of a vitamin. Duchenne is the most common and severe form of muscular dystrophy. Because of this genetic disease, one out of every 3,500 children spends their 12th birthday in a wheelchair. This disorder progressively leads to general paralysis, and most patients die of respiratory failure. The disease is caused by a genetic mutation that prevents a protein required to keep muscle cells intact from being produced. While most research focuses on repairing the defective gene, researchers at EPFL have come up with a different strategy.

Forty-four protein biomarkers discovered for Duchenne muscular dystrophy

Davis: Researchers have identified 44 proteins in the blood of patients with Duchenne muscular dystrophy that occur at significantly different concentrations in comparison to healthy individuals of the same age. Some protein biomarkers increase with disease progression, while others decrease. These biomarkers are expected to be useful to researchers and clinicians to study physiological processes of this rare muscle disease, as well as to monitor treatment progress and develop more effective therapies and diagnostic tools.

Bacterial Defense Mechanism Targets Duchenne Muscular Dystrophy

Duke University. US: Gene therapy approach could treat 60 percent of Duchenne Muscular Dystrophy patients. Duke researchers have demonstrated a genetic therapeutic technique that has the potential to treat more than half of the patients suffering from Duchenne Muscular Dystrophy (DMD).

Towards a new weapon against muscular dystrophy

CNRS. France: Research efforts associating scientists from the CNRS, UVSQ and INSERM within the Laboratoire END-ICAP1, working in collaboration with a team from the University of Bern, has demonstrated the therapeutic potential of a new class of synthetic oligonucleotides2 in the treatment of Duchenne muscular dystrophy (DMD) using RNA "surgery".

New study finds UK failing adults with Duchenne muscular dystrophy

Newcastle University. UK: Adults in the UK with Duchenne muscular dystrophy are being failed on critical medical checks that could lengthen their lives, according to research published by Newcastle University.

A study across seven EU countries found that despite investing in specialist care for children with Duchenne muscular dystrophy, the NHS is failing to meet internationally agreed standards on life-extending care for many adults with the condition, including vital annual heart and lung checks.

Stem cells faulty in Duchenne muscular dystrophy, researchers find

Stanford University. US: : In a mouse model of Duchenne muscular dystrophy, muscle stem cells express connective-tissue genes associated with fibrosis and muscle weakness, according to a new study.

Like human patients, mice with a form of Duchenne muscular dystrophy undergo progressive muscle degeneration and accumulate connective tissue as they age. Now, researchers at the Stanford University School of Medicine have found that the fault may lie at least partly in the stem cells that surround the muscle fibers.

Duchenne muscular dystrophy

Orphanet: Duchenne muscular dystrophy (DMD) is a neuromuscular disease characterized by rapidly progressive muscle weakness and wasting due to degeneration of skeletal, smooth and cardiac muscle.

DMD primarily affects males with an estimated incidence of 1/3,300 male births. Females are usually asymptomatic but a small percentage of female carriers manifest milder forms of the disease (symptomatic form of muscular dystrophy of Duchenne and Becker in female carriers).