Duchenne muscular dystrophy

Product

Last mentioned: Mar 10, 2026

Timeline

  1. Document Request

    Expected date for formal requests for internal FDA communications regarding DMD drug applications.

  2. Investigation Announced

    Sen. Ron Johnson publicly declares a formal inquiry into the FDA's rare disease drug approval process.

  3. Commissioner Consultation

    Johnson speaks with FDA Commissioner Marty Makary to discuss specific drug rejections and agency policy.

Stories mentioning Duchenne muscular dystrophy 1

regulation Neutral

Senator Johnson Launches Investigation into FDA Rare Disease Drug Denials

Senator Ron Johnson (R-Wis.) has initiated a formal investigation into the FDA’s regulatory processes regarding the rejection of biologic therapies for Duchenne muscular dystrophy and other rare diseases. The probe follows high-level discussions with FDA Commissioner Marty Makary and focuses on whether the agency's evidentiary standards are too restrictive for life-threatening conditions.

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