Wednesday, 19 April 2017

Researchers correct Duchenne muscular dystrophy using gene-editing alternative

Researchers correct Duchenne muscular dystrophy using gene-editing alternative: "Using the new gene-editing enzyme CRISPR-Cpf1, researchers at UT Southwestern Medical Center have successfully corrected Duchenne muscular dystrophy in human cells and mice in the lab.

The UT Southwestern group had previously used CRISPR-Cas9, the original gene-editing system, to correct the Duchenne defect in a mouse model of the disease and in human cells. In the current work, they used a new variation of the gene-editing system to repair the defect in both a mouse model and in human cells."



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Wednesday, 8 March 2017

Early treatment with heart failure drug can improve cardiac function in young boys with DMD

Early treatment with heart failure drug can improve cardiac function in young boys with DMD: "Researchers at The Ohio State University Ross Heart Hospital and Nationwide Children's Hospital have shown early treatment with the heart failure medication eplerenone can improve heart function in young boys with Duchenne muscular dystrophy (DMD) and stabilize heart function in older boys with the disease."



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Monday, 13 February 2017

FDA approves new treatment for wide range of patients with Duchenne muscular dystrophy

FDA approves new treatment for wide range of patients with Duchenne muscular dystrophy: "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."



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Thursday, 5 January 2017

What You Need to Know about Duchenne

What You Need to Know about Duchenne: "Viral gastrointestinal (GI) viruses are no fun for anyone, but they are especially worrisome for a person living with Duchenne muscular dystrophy. GI viruses affect the GI track – the stomach and intestine (i.e., ‘gut’) – resulting in abdominal pain/discomfort, nausea, vomiting, intestinal pain/discomfort, cramps and diarrhea. Thank goodness they usually have a ‘short course,’ most lasting less than 24 hours."

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Wednesday, 16 November 2016

Immune system plays important role in Duchenne muscular dystrophy, research reveals

Immune system plays important role in Duchenne muscular dystrophy, research reveals: "A new paper, co-written by faculty at Binghamton University, State University of New York, increases the understanding of Duchenne muscular dystrophy (DMD)—one of the most common lethal genetic disorders—and points to potential therapeutic approaches.

"The findings suggest that the immune system has an important role in the muscle disease of Duchenne muscular dystrophy," said Eric Hoffman, professor of pharmaceutical sciences and associate dean for research at Binghamton University's School of Pharmacy and Pharmaceutical Sciences."

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» GTx exploring product candidate as potential Duchenne treatment Action Duchenne

» GTx exploring product candidate as potential Duchenne treatment Action Duchenne: "GTx is exploring SARM, their lead product candidate, a selective androgen receptor modulator as potential treatment for Duchenne muscular dystrophy. 

Preclinical studies have confirmed the beneficial effects from SARMs in mice genetically altered to simulate Duchenne muscular dystrophy, compared to control groups. The Company continues to pursue a potential strategic collaboration with biopharma companies experienced in orphan drug development."

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