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Accelerating Therapy Development
Action Alert

We are committed to working with key stakeholders, including the US Food and Drug Administration (FDA), to help accelerate the development of life-changing therapies. Below are our priorities to accelerate therapeutic development.

MDA's Accelerating Therapy Development Priorities

Advocate for innovative ways to accelerate therapeutic development and FDA regulatory review so patients can gain access to life-changing treatments sooner.

  • Ensure the successful implementation of the many FDA and clinical trial reforms MDA advocates helped pass in 2022.
  • Ensure the continued success of the Orphan Drug Act, which has led to many vital therapies for rare diseases.
  • Ensure the FDA hears from the neuromuscular community perspective and to gain an appreciation for the factors that are taken into account by patients when a treatment is chosen.
  • Ensure the federal government continues to invest in medical research..
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