MUSCULAR DYSTROPHY ASSOCIATION, INC.

Medical/Disease Research/Clinical Labs

Advocated in favor of robust appropriations funding for the National Institutes of Health, the Food and Drug Administration, the Health Resources and Services Administration, and the Centers for Disease Control and Prevention. Advocated for greater appropriations for the National Center for Advancing Translational Sciences (NCATS) Advocated for greater appropriations for the National Institutes of Health. Advocated for appropriations for HHS, NIH, and FDA to implement the ACT for ALS. Advocated for the passage of the Newborn Screening Saves Lives Reauthorization Act, H.R. 482 / S. 350, to encourage investment in the national newborn screening program in order to detect more neuromuscular conditions early to speed appropriate interventions for patients. Supported the passage of the e Diversifying Investigations Via Equitable Research Studies for Everyone (DIVERSE) Trials Act (H.R. 5030/S. 2706) Advocated for reforms to the FDA Accelerated Approval program as part of the end-of-year omnibus legislation. Advocated for the creation of an FDA regulatory pathway for diagnostics, including lab-developed tests and invitro diagnostics. Advocated for the passage of the RARE Act (S. 4185) as part of the Senate FDA User Fee process. Advocated for certain provisions within the Cures 2.0 Legislation (H.R.6000) Advocated for passage of FDA and clinical trial reforms within an end-of-year bill, including accelerated approval reforms, clinical trial diversity advancements, the RARE Act, reauthorization of the Orphan Product Grants Program, and more.

TargetedSENATE · HOUSE OF REPRESENTATIVES · Health & Human Services, Dept of (HHS) · Food & Drug Administration (FDA) · Natl Institutes of Health (NIH)

Pharmacy

Advocated for funding and implementation of the ACT for ALS, a law that should accelerate development of treatments for individuals with ALS and other neuromuscular diseases. Advocated for the passage of the Diversifying Investigations Via Equitable Research Studies for Everyone (DIVERSE) Trials Act (H.R. 5030/S. 2706) Supported passage of the Ending the Diagnostic Odyssey Act Advocated for the passage of the Accelerating Kids Access to Care Act Advocated for certain provisions within the Cures 2.0 Legislation (H.R.6000) Support the passage of the Precision Medicine for Children Act Advocated for reforms to the FDA Accelerated Approval program as part of the end-of-year omnibus package Advocated for the creation of an FDA regulatory pathway for diagnostics, including lab-developed tests and invitro diagnostics. Advocated for passage of FDA and clinical trial reforms within an end-of-year bill, including accelerated approval reforms, clinical trial diversity advancements, the RARE Act, reauthorization of the Orphan Product Grants Program, and more.