
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
Patient Worthy Posts on Homocystinuria

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

HCU Network America Conference Wrap Up!
The inaugural Homocystinuria Network America Conference was held April 21st-22nd in Westford, MA. The families in attendance traveled from all across the US for the

An October Rare Disease Event Just For You!
Want an excuse to get to Florida and learn more about the science of human genetics in society and health? Then we definitely have an

Register for this September International Congress of Inborn Errors of Metabolism Event TODAY!
August 21st is the online registration deadline for this rare disease event! The site says the 15th and the 21st, so either way, you’ll want