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BioWorld - Wednesday, April 8, 2026
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Home » Experts: Regulatory roadblocks stalling rare disease therapies
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Experts: Regulatory roadblocks stalling rare disease therapies

March 3, 2026
By Mari Serebrov
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A lot of distance lies between talking regulatory flexibility and actually being flexible. That message was driven home again after Uniqure NV disclosed in its latest earnings report March 2 that the U.S. FDA wants a sham-controlled study before it will consider approval of the company’s gene therapy AMT-130 in Huntington’s, a rare disease currently affecting about 41,000 people in the U.S.
BioWorld Regulatory Neurology/psychiatric Rare disease Gene therapy U.S. FDA Orphan drug

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