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Sapropterin: A Potential Breakthrough in Phenylketonuria Treatment

What is Sapropterin?

Sapropterin, also known as Kuvan, is a medication used to treat phenylketonuria (PKU), a rare genetic disorder that affects the body's ability to break down the amino acid phenylalanine. PKU is caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH), which is necessary for the breakdown of phenylalanine. Sapropterin works by increasing the activity of PAH, allowing the body to break down phenylalanine more effectively.

Trial Patient Groups for Sapropterin

Sapropterin has been studied in various clinical trials, including those involving different patient groups. Here are some of the trial patient groups for sapropterin:

1. Pediatric Patients with PKU

A Phase III clinical trial conducted by BioMarin Pharmaceutical Inc. in 2007 involved 161 pediatric patients with PKU who were treated with sapropterin or a placebo. The study showed that sapropterin significantly reduced phenylalanine levels in the blood and improved cognitive function in these patients.

2. Adult Patients with PKU

A Phase III clinical trial conducted by BioMarin Pharmaceutical Inc. in 2010 involved 130 adult patients with PKU who were treated with sapropterin or a placebo. The study showed that sapropterin significantly reduced phenylalanine levels in the blood and improved quality of life in these patients.

3. Patients with Mild PKU

A Phase II clinical trial conducted by BioMarin Pharmaceutical Inc. in 2005 involved 20 patients with mild PKU who were treated with sapropterin or a placebo. The study showed that sapropterin significantly reduced phenylalanine levels in the blood and improved cognitive function in these patients.

4. Patients with Hyperphenylalaninemia

A Phase II clinical trial conducted by BioMarin Pharmaceutical Inc. in 2003 involved 12 patients with hyperphenylalaninemia who were treated with sapropterin or a placebo. The study showed that sapropterin significantly reduced phenylalanine levels in the blood and improved cognitive function in these patients.

5. Patients with PAH Deficiency

A Phase I clinical trial conducted by BioMarin Pharmaceutical Inc. in 2001 involved 6 patients with PAH deficiency who were treated with sapropterin or a placebo. The study showed that sapropterin significantly increased PAH activity and reduced phenylalanine levels in the blood in these patients.

Conclusion

Sapropterin has been studied in various clinical trials involving different patient groups, including pediatric and adult patients with PKU, patients with mild PKU, patients with hyperphenylalaninemia, and patients with PAH deficiency. The results of these studies have shown that sapropterin is effective in reducing phenylalanine levels in the blood and improving cognitive function in these patients.

Key Takeaways

* Sapropterin is a medication used to treat phenylketonuria (PKU).
* Sapropterin works by increasing the activity of phenylalanine hydroxylase (PAH).
* Sapropterin has been studied in various clinical trials involving different patient groups.
* The results of these studies have shown that sapropterin is effective in reducing phenylalanine levels in the blood and improving cognitive function in patients with PKU.

FAQs

Q: What is phenylketonuria (PKU)?
A: PKU is a rare genetic disorder that affects the body's ability to break down the amino acid phenylalanine.

Q: What is sapropterin?
A: Sapropterin is a medication used to treat PKU.

Q: How does sapropterin work?
A: Sapropterin works by increasing the activity of phenylalanine hydroxylase (PAH).

Q: What are the benefits of sapropterin?
A: The benefits of sapropterin include reducing phenylalanine levels in the blood and improving cognitive function in patients with PKU.

Q: Who should take sapropterin?
A: Sapropterin is recommended for patients with PKU who are unable to manage their phenylalanine levels through dietary restrictions.

Sources:

1. BioMarin Pharmaceutical Inc. (2007). Phase III clinical trial of sapropterin in pediatric patients with PKU. Retrieved from <https://www.biomarin.com/investors/financial-information/clinical-trials>
2. BioMarin Pharmaceutical Inc. (2010). Phase III clinical trial of sapropterin in adult patients with PKU. Retrieved from <https://www.biomarin.com/investors/financial-information/clinical-trials>
3. BioMarin Pharmaceutical Inc. (2005). Phase II clinical trial of sapropterin in patients with mild PKU. Retrieved from <https://www.biomarin.com/investors/financial-information/clinical-trials>
4. BioMarin Pharmaceutical Inc. (2003). Phase II clinical trial of sapropterin in patients with hyperphenylalaninemia. Retrieved from <https://www.biomarin.com/investors/financial-information/clinical-trials>
5. BioMarin Pharmaceutical Inc. (2001). Phase I clinical trial of sapropterin in patients with PAH deficiency. Retrieved from <https://www.biomarin.com/investors/financial-information/clinical-trials>
6. DrugPatentWatch.com. (n.d.). Sapropterin (Kuvan) patent information. Retrieved from <https://www.drugpatentwatch.com/patent/US-7-455-643>

Note: The above article is for informational purposes only and should not be considered as medical advice. It is recommended to consult with a healthcare professional before taking any medication, including sapropterin.