miércoles, 4 de diciembre de 2019

In Huntington's, hope meets the hurdle of clinical trials

The Readout
Damian Garde

In Huntington's, hope meets the hurdle of clinical trials

When an experimental therapy for a currently untreatable disease reaches clinical trials, there's excitement and hope. But there's also desperation among patients who don't meet the criteria to enroll.

Such is the case with a clinical trial now underway to test a Roche treatment for Huntington's disease. The drug, called RG6042, is aimed at the genetic roots of the disease, and if it's successful, it could be life-changing for those affected by the genetic disorder. As with all trials, though, Roche's can only include so many people. So some Huntington's patients — those who are too old, too young, have other health conditions, or just couldn't get a spot — have been turned away from participating.

STAT's Andrew Joseph has the story of a trial that for now looks like the best, and really the only, option available to these patients, with multimedia from Matthew Orr and Dom Smith.

Read more.

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