Nitisinone

Introduction

Nitisinone is a prescription medicine primarily used to treat a rare genetic disorder known as hereditary tyrosinemia type 1 (HT-1). It works by reducing the formation of toxic substances in the body that can cause severe liver and kidney damage. Nitisinone helps prevent complications such as liver failure, kidney problems, and neurological crises in individuals with HT-1. It must be used under strict medical supervision and alongside a special diet low in tyrosine and phenylalanine.

Uses of Nitisinone

• Treatment of Hereditary Tyrosinemia Type 1 (HT-1)
• Prevention of liver and kidney damage caused by tyrosine buildup

How Nitisinone works

Nitisinone works by blocking the enzyme 4-hydroxyphenylpyruvate dioxygenase (HPPD), which plays a role in breaking down the amino acid tyrosine. By inhibiting this enzyme, Nitisinone reduces the accumulation of harmful byproducts, thus preventing damage to organs such as the liver and kidneys.

Benefits of Nitisinone

• Reduces toxic substance buildup in the body
• Prevents liver and kidney complications
• Improves overall quality of life in HT-1 patients
• Allows better disease management when combined with dietary restrictions

How to take Nitisinone

Nitisinone should be taken exactly as prescribed by your doctor. It is usually taken once daily, with or without food. The dosage is based on body weight and medical condition. It’s important to follow a low-tyrosine and low-phenylalanine diet during treatment. Do not stop taking the medicine without consulting your healthcare provider.

Type of Dosage Available

• Capsules
• Oral Suspension

Side effects of Nitisinone

• Eye pain or irritation
• Photophobia (light sensitivity)
• Increased tyrosine levels in blood
• Dry skin or rashes
• Low platelet count (rare)

Safety Advice

• Use under medical supervision only
• Regular blood tests required to monitor tyrosine levels
• Follow a strict diet recommended by your doctor
• Avoid during pregnancy unless advised by a doctor
• Do not consume alcohol while taking Nitisinone

Frequently Asked Questions (FAQs)

Q: What is Nitisinone used for?

A: Nitisinone is used to treat hereditary tyrosinemia type 1 (HT-1), a rare genetic condition that affects the liver and kidneys.

Q: Can I take Nitisinone without food?

A: Yes, Nitisinone can be taken with or without food. Follow your doctor’s specific instructions.

Q: Are there any dietary restrictions while on Nitisinone?

A: Yes, a low-tyrosine and low-phenylalanine diet is essential for effective treatment with Nitisinone.

Q: Can Nitisinone be used in children?

A: Yes, it is approved for use in both children and adults diagnosed with HT-1. Dosage is weight-dependent.

Q: Is long-term use of Nitisinone safe?

A: Long-term use is generally safe under medical supervision, with regular monitoring and dietary management.