Aqneursa

Niemann-Pick disease type C (NPC) is a genetic condition that’s caused by the body not being able to store cholesterol and other fats properly. As a result, they build up in different parts of the body, including the brain. This can lead to a variety of symptoms, including enlarged spleen or liver, difficulty speaking, trouble walking, and seizures. Symptoms typically worsen over time. The main way to treat NPC is by managing the symptoms. People who have NPC usually see multiple healthcare professionals to help. For example, they might work with occupational or physical therapists regularly to maintain or improve the ability to swallow and move around. Aqneursa (levacetylleucine) is one of two medications that’s FDA-approved to treat the neurological symptoms of NPC .

There’s no cure for NPC, but Aqneursa (levacetylleucine) may help with the certain symptoms of this condition. A study showed that people who take Aqneursa (levacetylleucine) might have some improvement with speaking, walking, and other movements. In general, treatments for NPC focus on easing the symptoms of this condition.

Ask your healthcare team how long you should take Aqneursa (levacetylleucine). Currently, guidelines for NPC don’t recommend a specific length of time to take this medication. But people who took Aqneursa (levacetylleucine) in the clinical study received treatment for about 3 months. So your healthcare team may ask you to try this medication for at least 3 months as well to see if it works for you. But they might ask you to stop this medication earlier if the side effects, such as vomiting or stomach pain, are too severe. Speak with your healthcare team before making any changes to your medication.

In a clinical study, researchers looked at how well Aqneursa (levacetylleucine) worked by scoring people based on how well they can do certain activities, including walking, sitting, and speaking. Results showed that when people took Aqneursa (levacetylleucine) for 3 months, they had more improvement in symptoms than when they took placebo. Though more research is needed, another study suggests that some people might experience improvements after taking Aqneursa (levacetylleucine) for 6 weeks. Ask your healthcare team about what you might expect after starting treatment.

If you experience vomiting after taking Aqneursa (levacetylleucine), let your healthcare team know. They'll talk with you about the next steps depending on how severe your symptoms are and how often you're vomiting. In general, vomiting raises your risk of losing too much fluid, which can cause you to become dehydrated and experience dizziness or fainting. Your care team will recommend ways for you to stay hydrated, such as drinking water and sports drinks to replenish fluids and electrolytes in your body. But if you continue experiencing nausea or vomiting and your symptoms make it difficult to hold down food or medication, your healthcare team might talk with you about other treatment options.