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Senator Johnson Investigates FDA Denials of Rare Disease Treatments

3/11/2026

47 10 Full Breakdown

Summary

Story summary

Senator Ron Johnson (R-Wis.) has launched an investigation into the Food and Drug Administration's (FDA) denials of treatments for rare diseases.
He plans to request complete response letters from FDA.
He may call FDA officials to testify about uniQure's Huntington's therapy and related trial demands.
The investigation follows the withdrawal of PTC Therapeutics' Translarna for Duchenne muscular dystrophy after FDA feedback.

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Story summary

Senator Ron Johnson (R-Wis.) has launched an investigation into the Food and Drug Administration's (FDA) denials of treatments for rare diseases.
He plans to request complete response letters from FDA.
He may call FDA officials to testify about uniQure's Huntington's therapy and related trial demands.
The investigation follows the withdrawal of PTC Therapeutics' Translarna for Duchenne muscular dystrophy after FDA feedback.