MDA 2015 Scientific Conference

March 11-14, 2015- MDA Annual Scientific Conference in Washington, D.C., USA

The Muscular Dystrophy Association MDA is committed to enhancing the communication of new research findings and of information relating to the delivery of effective medical care for neuromuscular diseases. To achieve this goal, MDA hosts an annual conference series, with scientific and clinical conferences held in alternate years.  

Jain Foundation Attendees: Brad Williams

What is Stem Cell Therapy and can it be used to treat LGMD2B?

Stem cell therapy has been mentioned as a therapeutic option for a diverse group of diseases such as cancer, Parkinson, and muscular dystrophy.  Stem cell therapy involves introducing stem cells into patients to replace damaged tissues.  There are two main kinds of stem cells: embryonic and adult.  Embryonic stem cells are only found in developing embryos and these cells can develop into any cell type in the human body.

What is Exon Skipping and can it be used to treat LGMD2B?

Genes are the instructions manuals that tell the cell how to make the proteins that are needed for the cell to function properly.   When there is an error in those instructions (i.e. a mutation in the gene), the cell is unable to make its corresponding product (i.e. the protein) and this results in a disease.    If the part of the gene containing the error can be skipped, a slightly smaller but potentially functional protein could be made.

What is Gene Therapy and can it be used to treat LGMD2B?

Gene therapy is a therapeutic technique for genetic diseases, such as muscular dystrophies, that involves the injection of a patient with a gene carrier called a “vector.” The vector delivers to the patients’ cells a normal copy of the mutated gene to replace the protein whose absence causes the disease.    Gene therapy is a very attractive therapeutic possibility for the treatment of dysferlinopathy (i.e.

New Directions 2016 in Orlando, Florida, USA

June 29-July 2, 2016 - New Directions in Biology Conference in Orlando, FL, USA

The New Directions in Biology and Disease of Skeletal Muscle Conference highlighted recent developments in muscle biology, disease, and therapy with presentations by leading international researchers. It will be held in Orlando, Florida from June 29th to July 2nd, 2016.

Jain Foundation Attendees: TBD

October 4-8, 2016 - WMS in Granada, Spain

October 4-8, 2016 - WMS in Granada, Spain

The International Congress of the World Muscle Society is an annual international Congress on neuromuscular disorders. This year´s focus will be on Protein Aggregation, Autophagy and Proteomics, Limb-Girdle Muscular Dystrophies (LGMDs) and the Advances in Treatment of Neuromuscular Disorders.

For more information, visit their website here.

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