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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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?

Achieving adequate Phe control

Adherence to medication + diet

Cognitive and emotional impacts

Social/quality of life burdens

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

<20%

20%-50%

50%-80%

>80%

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

No patients

Some patients

Most of my patients

All of my patients

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

Lack of durable response

Adherence issues (treatment or dietary)

Adverse reactions

Residual cognitive and emotional impacts

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

Inadequate Phe control

Treatment adherence difficulty

Adverse reactions

Underestimating disease impact

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

Elevated Phe levels (>360 μmol/L)

Poor dietary adherence

Treatment adverse reactions

Neuropsychological symptoms

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

Saproterin

Palynziq

Diet or formula

Other

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Share insights

Ready to
rethink
PKU
control with PTC?

Join the
conversation