FDA defends its demand for new trial for rare brain disease therapy

A senior FDC official said the product is a “failed therapy” and confirmed that regulators have asked uniQure to run a new ...
Investing.com South Africa

uniQure downgraded after FDA backs sham-controlled trial for Huntington’s therapy

Investing.com -- Analysts downgraded uniQure N.V. after the U.S. Food and Drug Administration signalled it would require a sham surgery-controlled Phase 3 trial for the company’s Huntington’s disease ...
PharmExec

uniQure and FDA Clash Over Regulatory Decision for Huntington’s Disease Therapy: Report

The agency pointed to what it sees as a contradictory signal within uniQure's own dataset. Sham-controlled data from a small cohort of U.S. patients showed no treatment effect at 12 months, a finding ...
News-Medical.Net on MSN

New global guidelines improve reporting of pediatric randomized controlled trials

Researchers at The Hospital for Sick Children (SickKids), working with international collaborators and youth and family ...