Quest Magazine

Trial of Tadalafil in DMD Open to Participants

DMD/BMD: Stop Codon Read-Through Drug Trial Still Recruiting Participants

Blood-Filtering Procedure Removes Antibodies That Impede Gene Transfer

A research group supported in part by MDA has found that a blood-filtering strategy known as plasmapheresis looks promising as a way to help overcome one type of unwanted immune response to gene transfer therapy. Gene transfer therapy is the addition of therapeutic genes to treat disease, and it is often administered via transport vehicles made from viruses.

Louis Chicoine at Nationwide Children's Hospital and Ohio State University in Columbus, Ohio, and colleagues, reported the findings online Oct. 23, 2013, in the journal Molecular Therapy.

MDA Fights Back Against Muscle Disease, Launches 2014 Muscle Walk Season On #GIVINGTUESDAY

Study Seeks Parents, Guardians of Young Children with Neuromuscular Disease

The National Institute of Nursing Research, part of the National Institutes of Health (NIH) in Bethesda, Md., is seeking parents or guardians of children from birth through age 5 with a neuromuscular disease for a study to develop a questionnaire about motor function in young children.

Survey for People with IBM Reports Early Findings

Investigators conducting an online survey launched in January 2013 to gather anonymous information from people with inclusion-body myositis (IBM) are now reporting preliminary results and are asking the original respondents to complete a short supplementary survey.

ALS Nuedexta Study Seeks 60 Participants

The Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) is actively seeking people with amyotrophic lateral sclerosis (ALS) to participate in a phase 2 clinical trial designed to evaluate whether Nuedexta has any effects on bulbar functions including speech, swallowing and saliva function.

Painting by Louisiana Artist Accepted into MDA Art Collection

DMD/BMD: Summit and University of Oxford to Collaborate on Drug Development

MDA-supported biotechnology company Summit PLC has entered into a collaboration with the University of Oxford (United Kingdom) for continued development of utrophin modulators to treat Duchenne muscular dystrophy (DMD) and possibly Becker muscular dystrophy (BMD).

Abnormal Immune Response to LRP4 Protein Can Cause MG

A research team supported in part by MDA has shown that an abnormal reaction of the immune system against a protein called LRP4 can be added to the known causes of myasthenia gravis (MG), a disorder involving fluctuating weakness and fatigue because of impaired nerve-to-muscle communication. MG is an autoimmune disease, meaning it's caused by abnormal activity of the body's immune system against its own tissues.

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