Navigation Links
Nationwide Children's Hospital involved in expanded access program for treatment of PKU
Date:11/5/2007

Nationwide Childrens Hospital announced today its involvement in an expanded access program for sapropterin dihydrochloride, or sapropterin, an investigational treatment for phenylketonuria (PKU), a rare genetic metabolic disorder that, if left unmanaged in infants and children, can result in severe mental retardation. Currently, there are eight patients enrolled in the expanded access program at Nationwide Childrens.

Under an expanded access program, the U.S. Food and Drug Administration (FDA) allows early access to drugs that are being developed to treat serious diseases based on certain circumstances.

Individuals with PKU are born with a deficient enzyme called phenylalanine hydroxylase (PAH). With little or no PAH activity, they are unable to metabolize an essential amino acid, called phenylalanine (Phe), which is found in most foods including meat, eggs, dairy, nuts, pasta, bread and all fruits and vegetables. When Phe cannot be metabolized by the body, abnormally-high levels of it accumulate in the blood and are toxic to the brain, potentially causing brain damage that can result in severe mental retardation, seizures, tremors, phobias and general mental and behavioral decline. The only way that PKU patients can manage their disorder is by monitoring their daily intake of Phe; however, compliance with diet is a major problem for PKU patients due to limited food choices, the social limitations of following a strict diet, the poor taste of required formulas, and the availability and cost of specially-produced low-Phe foods.

By making sapropterin available through expanded access, we hope patients living with PKU will experience lower Phe levels and increased Phe tolerance, said Kim McBride, MD, MS an investigator in the Center for Molecular and Human Genetics at The Research Institute at Nationwide Childrens Hospital, and a faculty member at The Ohio State University College of Medicine. Since elevated Phe levels are toxic to the brain and can lead to severe health complications, we are encouraged there may be a new drug treatment designed to manage Phe levels in this patient population.

Patients with a confirmed diagnosis of hyperphenylalaninemia due to PKU who are older than 8 years of age, not currently enrolled in a clinical study with sapropterin, meet the inclusion criteria, and do not meet any of the exclusion criteria, may be eligible to participate in this program.


'/>"/>

Contact: Mary Ellen Fiorino
614-722-4595
Nationwide Children's Hospital
Source:Eurekalert

Related biology news :

1. Study finds environmental tests help predict hospital-acquired Legionnaires disease risk
2. Seattle Childrens Hospital leads $23.7 million NIH grant to study gene repair
3. 3 out of 4 hospital patients suffer from malnutrition regardless of their pathology
4. Researchers identify proteins involved in new neurodegenerative syndrome
5. New study shows greenback cutthroat trout involved in recovery effort misidentified
6. Silicon Valley Technology Leaders LaserCard Corporation and Tesla Motors Sign LaserPass Secure Access Deal
7. Mandate for public access to NIH-funded research poised to become law
8. UK research community gets extended access to ScienceDirect
9. IDEMA Reveals Program Highlights for DISKCON USA 2007
10. Thousands of starving children could be restored to health with peanut butter program
11. Scientists launch deep-sea scientific drilling program to study volatile earthquake zone
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:4/14/2016)... TEL AVIV, Israel , April 14, 2016 /PRNewswire/ ... in Behavioral Authentication and Malware Detection, today announced the ... has already assumed the new role. Goldwerger,s ... for BioCatch, on the heels of the deployment of ... In addition, BioCatch,s behavioral biometric technology, which discerns unique ...
(Date:3/31/2016)... RATON, Florida , March 31, 2016 ... LEGX ) ("LegacyXChange" or the "Company") ... for potential users of its soon to be launched ... video ( https://www.youtube.com/channel/UCyTLBzmZogV1y2D6bDkBX5g ) will also provide ... the use of DNA technology to an industry that ...
(Date:3/29/2016)... BOCA RATON, Florida , March 29, 2016 /PRNewswire/ ... ("LegacyXChange" or the "Company") LegacyXChange "LEGX" and SelectaDNA/CSI Protect ... Synthetic DNA in ink used in a variety of ... preventing theft. Buyers of originally created collectibles from athletes ... authenticity through forensic analysis of the DNA. ...
Breaking Biology News(10 mins):
(Date:4/26/2016)... ... April 27, 2016 , ... ... Rothgerber Christie LLP as an associate in the firm’s Intellectual Property practice group. ... mechanical and electromechanical patent applications. He has an electrical engineering and computer engineering ...
(Date:4/26/2016)... ... ... The European Patent Office (EPO) today announced that U.S. ... European Inventor Award 2016 in the category "Non-European countries." The winners of the 11th ... Lisbon on June 9th. , The human capacity to walk with fluidity is the ...
(Date:4/26/2016)... ... April 26, 2016 , ... Mr. Palmer created the RPO ... the first multi-million dollar, multi-year managed services contract in the U.S. intelligence community with ... our leadership team,” said John Younger, founder of Accolo. “We are growing and ...
(Date:4/26/2016)... , ... April 26, 2016 , ... BioFactura, Inc ... Series A round of financing. Healthy investor interest drove significant oversubscription of the ... generic biologics, known as biosimilars, to the advanced preclinical stages. , Chief Executive Officer ...
Breaking Biology Technology: