Gaucher Disease :: Novel Approach Targets an Inherited Disorder Gaucher disease

Using a quantitative high-throughput screening strategy, researchers at the National Institutes of Health (NIH) have identified three new classes of small molecules that may prove useful for treating Gaucher disease, an inherited disorder that disrupts a cell?s ability to break down and dispose of certain cellular waste products.

Gene :: FDA statement on Gene Therapy Clinical Trial

On July_24, 2007 the U.S. Food and Drug Administration (FDA) was informed by Targeted Genetics Corporation of Seattle about the death of a patient who received an investigational gene therapy product in a clinical trial for the treatment of active inflammatory arthritis.

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