Patients & Caregivers

Patients & Caregivers
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The challenges of having homocystinuria

The manifestations of homocystinuria continue to progress with the classic clinical symptoms reaching varying degrees of disability and impact on the quality of life of affected individuals. Regardless of the individual’s age of onset, the loss of biochemical control at any age is associated with the development of serious complications that can be life-threatening. Although a restricted diet decreases homocysteine levels in fully compliant patients to varying degrees, most patients require additional therapies to achieve their target homocysteine levels, which are still far above normal levels. Compliance with diet is often poor because of the complexities of the diet and its severe restrictions.

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“It’s a huge struggle to get kids to take formula. I hope for their sake there is (treatment) management that is more realistic.”

Patient resources

HCU Network AmericaNORDHCU Network Australia

We put patients first. While innovative research and development is our backbone, we take the time to listen to what the patients and medical community are telling us. Basic research advances the field but our focus is on bringing therapeutics to patients and improving patients’ lives as quickly as possible.