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Challenges of developing and conducting clinical trials in rare disorders

11 In the past, decompression surgery was an option to relieve stress on the facial nerve, but the benefits currently do not outweigh the risks, and permanent postoperative hearing loss has been reported in up to 15% of patients undergoing this procedure

MR cholangiopancreatography and endoscopic retrograde cholangiopancreatography have high specificity and sensitivity for detection of primary disease and assessment of disease progression

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