Up at 5AM: The 5AM Solutions Blog

Despite Cure, Leprosy Hangs On

Posted on Tue, Feb 17, 2015 @ 03:00 PM

The World Health Organization (WHO) has made safe, effective multidrug therapies (MDT) to cure leprosy Leprosy_deformities_handsavailable free of charge worldwide, and has been largely eliminated in the world. Largely, but not completely.

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Tags: Cure, rare diseases

Clinical Trials: Finding Goldilocks to Cure Rare Diseases

Posted on Tue, Dec 09, 2014 @ 03:00 PM

One of the challenges in drug development is enrolling participants in clinical trials that meet very specific criteria. The reason for this is sound: make the trial too broad and researchers risk missing meaningful data about a potential therapy's efficacy.

However, there is a tension between mitigating that risk and the need to reach as many people with horribly debilitating -- and often life-threatening -- illnesses as possible with a life-altering miracle.  Often multiple trials have to be conducted that test lots of different scenarios, and each one needs participants whose conditions, like the porridge and the bed in the fairy tale, are "just right."

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Tags: clinical trials, rare diseases, drug development

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