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"Truly evil" FDA rejection of gene therapy overturned after Trump official ousted

A gene therapy for Huntington's disease has a new path toward approval from the Food and Drug Administration after the ouster of several Trump officials, particularly Vinay Prasad, who rejected the therapy in a shocking move one former FDA official called "truly evil."

Huntington's disease is an inherited condition that typically strikes in middle age and causes nerve cells in the brain to gradually break down. There are currently no treatments for the disease, and many afflicted die in their 50s and 60s.

Gene therapy company UniQure developed a one-time treatment, AMT-130, that aims to lower brain levels of the mutant protein behind the disease, called huntingtin. Data from a small, early trial suggested the drug could slow the progression of the disease up to 75 percent, and patients and advocates have closely watched the drug's development in hopeful anticipation.

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Β© Getty | Congressional Quarterly

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Diabetes org apologizes for ejecting scientists over criticism of Trump

Amid intense backlash, the head of the American Diabetes Association posted a video Wednesday apologizing for the organization's decision on Friday to forcefully remove five leading diabetes scientistsΒ from the association's annual meeting.

The scientists were ejected for handing out copies of an April editorialβ€”published in the ADA's own journal Diabetes Careβ€”that sharply criticizes the Trump administration for the damage and destruction it's wreaking on biomedical research. The five scientists included Steven Kahn, professor of medicine at the University of Washington, who is the editor-in-chief of Diabetes Care and a co-author of the editorial. It also included former ADA President Desmond Schatz of the University of Florida.

The scientists were distributing the editorial outside the conference's opening speech, which was originally scheduled to be given by Jay Bhattacharya, head of the National Institutes of Health under Trump. Bhattacharya canceled at the last minute, and senior NIH official Rick Woychik took his place.

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Β© Screenshot/MedPage Today

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