New results for a #rare neuromuscular disease #clinical trial

In Uncategorized by Ana Lloyd

clinical trial, limb-girdle muscular dystrophy, rare disease, orphan drugs, World Orphan Drug Congress A rare disease Phase I clinical trial of gene therapy for limb-girdle muscular dystrophy type 2C presented encouraging results.

Clinical trials between December 2006 and December 2009 proved that the injections were well tolerated and not associated with adverse physical or biological effects. The results mean the treatment becomes efficacious which is very rare in any Phase I clinical trial.

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Clinical trials such as their ethical aspects, innovative methods for recruitment, improvement for research outcomes, and more will be discussed for the major institutions and its experts at the World Orphan Drug Congress USA 2012.

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