Urea Cycle Disorder Treatment Challenges I SeeUCDifferently

Having to balance treatment and the constant stress of elevated ammonia levels can be difficult, especially when treatment for urea cycle disorders (UCDs) brings its own obstacles. While many UCD patients and their caregivers try to follow their treatment plan to the best of their ability, it can be very challenging to take medicine as instructed—even when people’s lives depend on it. In fact, in one survey, 58% of doctors said patients were “compliant” (45%) or “very compliant” (13%) with taking their treatment.

58% of doctors said patients were “compliant” or “very compliant” with taking their treatment.

58% of doctors said patients were “compliant” or “very compliant” with taking their treatment.

Challenges of taking medication

Beyond keeping track of diet, ammonia levels, and how you or your loved one is feeling from day to day, there are more challenges that come with taking treatment. Attributes of treatments that can make them hard to take include:

Tolerability
(eg, stomach discomfort,
poor taste/odor)

Too many pills for urea cycle disorders — Administration
(eg, too many pills, frequent dosing)

Cost and access (including navigating insurance)

If you are having difficulty sticking to your or your loved one’s treatment plan, speak to the healthcare team about your concerns to help make sure ammonia levels are well managed.

Treatment options may help people with UCDs avoid some of the challenges of taking therapy. These could help people take their medication as prescribed and keep ammonia levels lower.

Treatment options may help people with UCDs avoid some of the challenges of taking therapy. These
could help people take their medication as prescribed and keep ammonia levels lower.

Even with treatment, long-lasting damage can still be a threat

Are you or a loved one currently taking a UCD treatment? Do you have trouble sticking to the treatment plan? If so, you are not alone. Many people with UCDs find it difficult to take their medication as prescribed and struggle to keep ammonia levels under control. This can be incredibly frustrating, as treatment challenges can lead to increased ammonia levels, resulting in HACs or worse.

Even mildly raised ammonia levels can be dangerous

Even small increases in ammonia can cause brain damage over time. This is important to know and monitor, as sometimes there are no noticeable symptoms of smaller ammonia increases. Treatment challenges can result in people not taking their medication as prescribed, which can lead to increased ammonia levels. Therefore, ways to overcome treatment challenges are urgently needed to avoid preventable brain damage and even death.

Doctors, patients, and caregivers alike see a need for additional treatment options, saying UCD treatment plans are not easily followed due to issues with:

Tolerability (eg, stomach discomfort, poor taste/odor)
Administration (eg, too many pills, frequent dosing)
Cost and access (including navigating insurance)

As research and understanding of UCDs and treatment challenges continue to develop, there is hope for more treatment options.

The need for additional treatments

Looking to the future of UCD treatments, there is hope for changes that could improve how UCDs are managed and lower the chances of increased ammonia levels and hyperammonemic crises (HACs).

The need for additional treatments

Looking to the future of UCD treatments, there is hope for changes that could improve how UCDs are managed and lower the chances of increased ammonia levels and hyperammonemic crises (HACs).

If you or a loved one is experiencing symptoms or needs help managing a UCD, talk to a
doctor as soon as possible.

Treatment challenges may result in increases in ammonia, as medication may not work properly when not taken as prescribed.

Overall, Treatment challenges may result in increases in ammonia, as medication may not work properly when not taken as prescribed.

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