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Not actual patients.

Ready to
rethink
PKU
control with PTC?

With 25 years of experience in rare diseases and genetic disorders, PTC Therapeutics is proud to extend our expertise to the PKU community. We understand the unique challenges of managing PKU, and together, we can reimagine PKU control for patients and their care partners.

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Sign up today to be among the first to receive updates from PTC Therapeutics about the future of PKU management. We look forward to working closely with you and joining your extended team.

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Share insights

Please take a moment to share your insights and experiences with managing PKU.

1Which of the following is the most significant challenge of managing PKU from a patient and care partner perspective?

2What percentage of your patients achieve and maintain blood Phe levels within the ACMG recommended range of 120‑360 μmol/L?

3How many of your patients achieve the ACMG’s secondary goals of therapy? (e.g., Phe tolerance, amelioration of symptoms, improved quality of life)

4Among patients who do respond to treatment, what difficulties have they experienced? (Select all that apply)

5In your experience, what is the primary reason a patient discontinues treatment?

6Which of the following comes to mind when considering inadequate PKU "control"? (Select all that apply)

7What is your preferred treatment choice for patients with PKU?

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US-PKU-0216 08/24

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