Investigate why the FDA is denying access to ataluren for patients with Duchenne muscular dystrophy.

I'm launching an investigation into why the FDA is denying access to a drug that has been helping patients with Duchenne muscular dystrophy for years.

Johnson commits to investigate the FDA's denial of access to ataluren for Duchenne patients.

Whistling Past the Graveyard - Ron Johnson Senator from Wisconsin
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The article says Ron Johnson said in March that he was investigating the FDA for rejections of rare disease treatments.

Recent reporting shows Johnson is still actively probing FDA rare-disease rejections, but it does not show a specific ataluren solution or access change.

unresolved same_term A for effort

'The FDA is a complete mess': Trump makes fate of agency chief unclear as public trust plummets
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Axios says Sen. Ron Johnson has been probing the FDA's rejection of rare disease drugs and that his concern dates back years.

This supports ongoing, serious oversight activity on rare-disease drug access, but it still falls short of evidence that ataluren access was delivered or resolved.

unresolved same_term A for effort

The cloud over the FDA has not yet lifted
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Within the June 9-July 8 lookback window, the report says FDA agreed to reverse a rejection of Regenxbio's rare-disease gene therapy and that the action was part of a broader series of rare-disease review reversals under new leadership. It does not identify ataluren, Translarna, Duchenne access, or a completed Johnson ataluren investigation.

Recent lookback-window activity shows FDA reconsidering other rare-disease rejections, but no concrete ataluren-specific delivery or access change.

unresolved same_term

FDA Gives Third Rare-Disease Drug Another Shot, Regenxbio Says
secondary · model gpt-5.5 · confidence 64%

Axios reports that Sen. Ron Johnson, chair of the Senate Permanent Subcommittee on Investigations, said he was probing FDA rejections of rare-disease drugs and had concerns dating back to 2017. The story frames this as ongoing scrutiny of FDA rare-disease decision-making, not as a completed ataluren-specific investigation or delivered patient access.

Johnson is taking concrete oversight action on FDA rare-disease rejections, but the source does not show ataluren-specific findings or access for Duchenne patients.

partial same_term A for effort

The cloud over the FDA has not yet lifted
secondary · model gpt-5.5 · confidence 78%

Congress.gov lists Sen. Ron Johnson as sponsor of S.204 and records that it became Public Law No. 115-176 on May 30, 2018. CRS summarizes the law as exempting provision of certain unapproved investigational drugs to terminally ill patients who exhausted approved options and cannot join a clinical trial.

Johnson delivered broader investigational-drug access legislation, a serious related effort, but it does not prove he completed the promised ataluren-specific FDA investigation.

partial same_term A for effort

S.204 - Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017
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EMA states that its human medicines committee confirmed a recommendation not to renew Translarna (ataluren) authorization for Duchenne muscular dystrophy because effectiveness had not been confirmed. EMA also notes Translarna had been authorized in the EU for DMD caused by nonsense mutation and that the review considered post-authorization studies, registries, patients, caregivers, clinicians, and organizations.

The key ataluren product remained scientifically and regulatorily unresolved internationally, reinforcing that no clear FDA access solution for U.S. Duchenne patients is shown.

unresolved same_term

Translarna: EMA re-confirms non-renewal of authorisation of Duchenne muscular dystrophy medicine
secondary · model gpt-5.5 · confidence 82%