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News & Resources

FDA Grants RASRx1902 Orphan Drug Status as Potential Therapy for Duchenne Muscular Dystrophy

The U.S. Food and Drug Administration has granted RASRx1902 orphan drug status to treat Duchenne muscular dystrophy (DMD), says its developer, RASRx of Newport Beach, California. The Orphan Drug status is given to therapies intended to prevent and treat rare life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people, and for which […]

Increasing Levels of Adiponectin Hormone Could Help Reverse Progression of Duchenne MD, Study Suggests

Increasing the levels of a muscle-inflammation-reducing hormone called adiponectin could help reverse the progression of Duchenne muscular dystrophy (DMD), a study suggests. The article in the American Journal of Pathology was titled “Potential Therapeutic Action of Adiponectin in Duchenne Muscular Dystrophy.” Adiponectin can reduce acute and chronic muscle inflammation. Previous studies have shown that increasing its expression […]

MicroRNA-29 overexpression by adeno-associated virus suppresses fibrosis and restores muscle function in combination with micro-dystrophin

Our overall goals include developing meaningful treatment for DMD boys at any stage of disease and one of the challenges to treat DMD is the accumulation of fibrosis or scar tissue as muscle loss progresses.  We have previously demonstrated a way to replace the missing dystrophin gene with a miniature dystrophin using gene therapy.  We […]

PTC Therapeutics Completes Acquisition of Emflaza™ for the Treatment of Duchenne Muscular Dystrophy in the U.S.

SOUTH PLAINFIELD, N.J., April 20, 2017 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced it has completed its acquisition of all rights to Emflaza™ (deflazacort) for the treatment of Duchenne muscular dystrophy (DMD) in the U.S. Execution of the asset purchase agreement setting forth the terms of the acquisition was announced on March 16, 2017. […]

Gene-editing alternative corrects Duchenne muscular dystrophy

DALLAS – April 12, 2017 – 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 […]

Duchenne treatment receives FDA clearance for clinical development

Akashi Therapeutics now have the greenlight from the FDA to continue clinical development of HT-100. The biopharmceutical company is developing treatments for patients suffering from Duchenne muscular dystrophy (DMD). HT-100 (delayed-release halofuginone) development will continue under the new study name HALO-DMD-04. Following the clearance from the FDA, Marc Blaustein, Chief Executive Officer at Akashi Therapeutics […]

PTC Therapeutics Announces FDA Acknowledgment of New Drug Application Filing for Translarna™ for the Treatment of Nonsense Mutation Duchenne Muscular Dystrophy

SOUTH PLAINFIELD, N.J., March 6, 2017 /PRNewswire/— PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the U.S. Food and Drug Administration (FDA) has acknowledged the filing over protest of PTC’s New Drug Application (NDA) for Translarna™ (ataluren), an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). The Company […]

EIM Could Be Reliable Method to Assess Effects of Therapy in DMD Trials

A technique called electrical impedance myography (EIM) can help detect muscle deterioration in people with Duchenne muscular dystrophy (DMD), according to a study published in the scientific journal Annals of Neurology. The method is also able to identify the therapeutic effects of corticosteroids and could therefore be a good biomarker in clinical trials. To evaluate […]

Translarna in Canada

Translarna received marketing authorization by the European Medicines Agency and is available in Europe and regions that reference that authorization. Currently, Translarna has not received marketing authorization in Canada. Based on the outcome of ongoing regulatory interactions we are developing a plan to file a New Drug Submission for Translarna in 2017. PTC remains committed […]

Scientists replace piece of gene mutated in Duchenne muscular dystrophy in effort to make healthy

AUGUSTA, Ga. (Dec. 19, 2016) – Scientists are using “gene scissors” to cut off the code of a defective gene that results in progressively weaker muscles and death in Duchenne muscular dystrophy and replace it with a synthetic code they hope will one day restore healthy life to these patients. “We want to use genetically […]

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