And Ford has bet heavily on Martens' strategy to develop multiple models from just a few global vehicle platforms.

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Should the drug make it to market, it might help many of the 14, 400 patients who develop multiple myeloma each year in the U.S. As it is, most die within five years.

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Ms. Buckley says she appreciates that multiple research groups are trying to develop treatments for Sanfilippo.

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