PTC THERAPEUTICS, INC.

Health Issues

Issues related to access to rare disease therapies Issues related to FDA modernization and rare disease drug development Issues related to the Accelerated Approval Program Issues related to Public Law No. 117-169 -The Inflation Reduction Act of 2022 Issues related to rare disease diagnosis and testing Issues related to alternative funding programs Issues related to the Rare Pediatric Disease Priority Review Voucher program, including H.R. 1262 - Give Kids a Chance Act of 2025 and S. 932 - Give Kids a Chance Act Issues related to Public Law No. 119-21 - The One Big Beautiful Bill Act of 2025

TargetedSENATE · HOUSE OF REPRESENTATIVES

Medical/Disease Research/Clinical Labs

Awareness of Aromatic L-amino acid decarboxylase deficiency (AADCD) Improving diagnostic testing for rare pediatric genetic diseases Awareness of Huntingtons Disease Awareness of Friedreichs Ataxia Awareness of Phenylketonuria Awareness of Duchenne Muscular Dystrophy

TargetedSENATE · HOUSE OF REPRESENTATIVES

Medicare/Medicaid

Innovative payment methodologies for cell and gene therapies Issues related to Medicaid access across state lines Issues related to Medicaid coverage of Accelerated Approval therapies Issues related to Huntingtons Disease disability insurance access Issues related to the Medicaid Drug Rebate Program Issues related to implementation of Public Law No. 117-169 - The Inflation Reduction Act of 2022, specifically the orphan drug exemption, including H.R. 946 - The Optimizing Research Progress Hope and New Cures Act and the small molecule penalty, including H.R. 1492 - The Ensuring Pathways to Innovative Cures Act