Summit Therapeutics PLC to receive US$22mln milestone payment as final patient enrols for DMD trial

Summit Therapeutics PLC to receive US$22mln milestone payment as final patient enrols for DMD trial: "Summit Therapeutics PLC (NASDAQ:SMMT LON:SUMM) will receive a US$22mln milestone payment from Sarepta Therapeutics (NASDAQ:SRPT) after the final patient was enrolled in a dosing trial of utrophin modulator, ezutromid.

Summit hopes the trial will identify utrophin modulation as a potential disease-modifying treatment for all patients suffering Duchenne Muscular Dystrophy (DMD),  a progressive and fatal muscle wasting disease that affects 50,000 boys and young men globally."



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Summit Therapeutics PLC to receive US$22mln milestone payment as final patient enrols for DMD trial

Summit Therapeutics PLC to receive US$22mln milestone payment as final patient enrols for DMD trial: "Summit Therapeutics PLC (NASDAQ:SMMT LON:SUMM) will receive a US$22mln milestone payment from Sarepta Therapeutics (NASDAQ:SRPT) after the final patient was enrolled in a dosing trial of utrophin modulator, ezutromid.

Summit hopes the trial will identify utrophin modulation as a potential disease-modifying treatment for all patients suffering Duchenne Muscular Dystrophy (DMD),  a progressive and fatal muscle wasting disease that affects 50,000 boys and young men globally."



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Preclinical data reinforces potential efficacy, durability of investigational gene therapy for DMD

Preclinical data reinforces potential efficacy, durability of investigational gene therapy for DMD: "Solid Biosciences announced today that new data from two preclinical studies reinforce the potential of its investigational microdystrophin gene therapy, SGT-001, to be an effective treatment approach for Duchenne muscular dystrophy (DMD). The preclinical data, which were presented at the American Society of Gene and Cell Therapy (ASGCT) 20th Annual Meeting, demonstrated that a single administration of SGT-001 resulted in sustained and significant microdystrophin expression and improvements in muscle function, with no observed immune response. The Company plans to initiate clinical trials for SGT-001 in the second half of 2017."



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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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Utrophin modulators significantly improve muscular dystrophy in the mdx diaphragm - Neuromuscular Disorders

Utrophin modulators significantly improve muscular dystrophy in the mdx diaphragm - Neuromuscular Disorders: "Utrophin modulators significantly improve muscular dystrophy in the mdx diaphragm"



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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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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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