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[Comment] Treatments for rare diseases: molybdenum cofactor deficiency

In The Lancet, Bernd Schwahn and colleagues1 report on the follow-up of a cohort of infants with molybdenum cofactor deficiency (MoCD), some of whom benefited greatly from a novel therapy. A very rare metabolic disorder is perhaps an unlikely subject for a general medical journal, but new treatments for rare metabolic diseases have problems in common that need much thought.

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