NIH expert sees new drive against rare disease

The Wall Street Journal's coverage of a research program to develop a therapy for progeria, an extremely rare disease which afflicts 42 known patients, includes a discussion with Steven Groft, the director of the NIH's office of rare diseases. The interview includes his ideas on how research in rare diseases is likely to grow internationally, with advocacy groups playing a critical role in spurring new research programs in search of a cure.

"In progeria (a disease that typically kills children by the age of 13) and other diseases," he says, "these groups provide a model for getting research completed that can lead to interventions. From what we have seen, the most successful product-development programs involve strong advocacy groups."

Groft centers on the need to get researchers interested in a rare disease and to follow that up by bringing biotech companies into play to develop a marketable product. As other countries begin to host new research programs into rare diseases, the number of therapies will grow as well.

- here's the Q&A from The Wall Street Journal (sub. req.)

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