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How Does AADC Deficiency Impact Patients and Their Families? 

PTC Therapeutics
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Most children with AADC deficiency have significantly shortened
lifespans. It’s a very disabling disorder. They don’t feed well. They can’t control their blood pressure and their temperature so they get uncontrolled fevers. When they’re sick, they can develop dystonic storming or uncontrolled dystonia, which can be life-threatening in and of itself. Like many other disorders that involve tone and development in children, AADC has a substantial impact on the family - not just the child, but really the whole family.
Caregivers, to learn more about AADC deficiency please visit our website aboutaadc.com/​
Healthcare professional interested in learning more? Visit aadcinsights.com/​
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About PTC Therapeutics, Inc.
PTC is a science-driven, global biopharmaceutical company focused on the discovery, development, and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need.

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20 апр 2021

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