» Sarepta Enters into Partnership with Flagship Biosciences to Digitally Automate the Measurement of Dystrophin, a Key Therapeutic Efficacy Marker for Muscular Dystrophy Action Duchenne

» Sarepta Enters into Partnership with Flagship Biosciences to Digitally Automate the Measurement of Dystrophin, a Key Therapeutic Efficacy Marker for Muscular Dystrophy Action Duchenne: "Sarepta Enters into Partnership with Flagship Biosciences to Digitally Automate the Measurement of Dystrophin, a Key Therapeutic Efficacy Marker for Muscular Dystrophy"



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New gene therapy protects mice from life-threatening heart condition

New gene therapy protects mice from life-threatening heart condition: "A new gene therapy developed by researchers at the University of Missouri School of Medicine has been shown to protect mice from a life-threatening heart condition caused by muscular dystrophy.

"This is a new therapeutic avenue," said Yi Lai, Ph.D., the leading author of the study and assistant research professor in the MU School of Medicine's Department of Molecular Microbiology and Immunology. "This is just a first step, but we hope this could lead to a treatment for people with this devastating heart condition, which is a leading cause of death for people with Duchenne muscular dystrophy.""



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Researchers use new gene editing method to correct mutation that leads to DMD

Researchers use new gene editing method to correct mutation that leads to DMD: "UT Southwestern Medical Center researchers successfully used a new gene editing method to correct the mutation that leads to Duchenne muscular dystrophy (DMD) in a mouse model of the condition.

Researchers used a technique called CRISPR/Cas9-mediated genome editing, which can precisely remove a mutation in DNA, allowing the body's DNA repair mechanisms to replace it with a normal copy of the gene. The benefit of this over other gene therapy techniques is that it can permanently correct the "defect" in a gene rather than just transiently adding a "functional" one, said Dr. Eric Olson, Director of the Hamon Center for Regenerative Science and Medicine at UT Southwestern and Chairman of Molecular Biology."



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» Translarna Press Release -First ever treatment for rare muscle wasting condition, Duchenne Muscular Dystrophy, given conditional approval by the European Commission Action Duchenne

» Translarna Press Release -First ever treatment for rare muscle wasting condition, Duchenne Muscular Dystrophy, given conditional approval by the European Commission Action Duchenne: "Translarna Press Release -First ever treatment for rare muscle wasting condition, Duchenne Muscular Dystrophy, given conditional approval by the European Commission"



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Splice-switching targeted oligonucleotide drugs alter editing of gene transcript

Splice-switching targeted oligonucleotide drugs alter editing of gene transcript: "In splice-switching, an innovative therapeutic approach, targeted oligonucleotide drugs alter the editing of a gene transcript to produce the desired form of a protein. Developments in this rapidly advancing field have already led to promising treatments for such diseases as Duchenne Muscular Dystrophy and spinal muscular atrophy, as described in an article in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available on the Human Gene Therapy website."



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Illinois Designates Week of June 23 as Duchenne Awareness Week! - PPMD Community

Illinois Designates Week of June 23 as Duchenne Awareness Week! - PPMD Community: "The State of Illinois is recognizing our 20th Annual Connect Conference in Chicago, Illinois, June 26-29, 2014 by declaring the week of June 23 as Duchenne Muscular Dystrophy Awareness Week! With over 500 families and Duchenne experts expected to attend from around the world, this will be our largest Annual Connect Conference to date."



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Webinar: Ataluren (Translarna) Update with PTC - PPMD Community

Webinar: Ataluren (Translarna) Update with PTC - PPMD Community: "In a webinar hosted by United Parent Project Muscular Dystrophy, Robert Spiegel, MD, FACP, chief medical officer of PTC Therapeutics, presented an overview of the positive opinion by the Committee for Medicinal Products for Human Use of the European Medicines Agency regarding the company’s application for a conditional marketing authorization of TranslarnaTM (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy, and what this decision means for families living in and outside the EU."



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