HCU Network America

The HCU Network America strives:

to inform and provide resources for patients and families, create connections, influence state and federal policy, and support advancement of diagnosis and treatment for Homocystinuria and related disorders.
to support research that improves diagnosis and treatment including a cure for the disease.
to create connections across the community and facilitate sharing of information and best practices through in-person and virtual events and discussions.
to assure all patients are diagnosed as early and efficiently as possible to enable access to care and avoid complications.
to provide information and resources to better manage the disease. Resources include:
- HCU treatment and dietary approaches
- General disease management
- Increased access for treatment and supplements
- Research findings and opportunities for clinical trials

Condition Awareness & Advocacy

Here is a list of conditions this partner raises awareness and advocacy for:

Resources & Support

Connect With HCU Network America

Patient Worthy Posts on Homocystinuria

HCU isn’t an allergy: Jessica’s Homocystinuria Story

When Jessica Karnes was a kid, she was told she was no longer allowed to eat chicken nuggets. As a self-proclaimed lifelong picky eater—and a four-and-a-half-year-old

A Clinical Trial Now Testing a Novel, Engineered Enzyme for Patients with Classical Homocystinuria

In a recent article appearing in BioPortfolio, Orphan Technologies announced a Phase 1/2 clinical trial involving the first Classical Homocystinuria (HCU) patients to be treated with OT-58.

Yeast May be the Key to Uncovering Treatments for Homocystinuria and Other Metabolic Conditions

There is a great unmet medical need for patients living with metabolic disorders. There are few approved treatments for these conditions and many are fatal.

Homocystinuria is More Common Than We Thought, It’s Just Not Detected Well in NBS

Most patients diagnosed with Homocystinuria (HCU), a rare metabolic disorder, don’t receive their diagnosis until later in life. Scientists were at a loss for why