Navigation Links
Meta-Analysis of Blood Phe Levels and Clinical Outcomes in PKU Published
Date:9/27/2007

NOVATO, Calif., Sept. 27 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that a systematic literature review and meta-analysis of blood phenylalanine (Phe) levels and clinical outcomes in phenylketonuria (PKU) was published in the September/October 2007 issue of Molecular Genetics and Metabolism. The review of 40 studies with within-study correlations between blood Phe level and intelligence quotient (IQ) confirms a significant correlation between blood Phe level and IQ in PKU patients and supports the use of blood Phe levels as a predictive biomarker for IQ in clinical trials.

"This meta-analysis stresses the importance of early and continuous Phe control to prevent irreversible neurological damage in PKU patients and importantly, supports the use of blood Phe levels as the primary endpoint in our Kuvan clinical trials," said Emil Kakkis, M.D., Ph.D., Chief Medical Officer of BioMarin. "We are working closely with the FDA in the final months leading up to the PDUFA date and look forward to providing the first drug treatment option to PKU patients by the end of the year, pending FDA approval."

Highlights from the meta-analysis are summarized below:

-- Significant, proportional correlations were found during critical

periods (0 to 12 years of age) for early treated patients with PKU

(r=-0.35; 95% confidence interval), where each 100 umol/l increase in

blood Phe levels predicted an average 1.3 to 3.1 point reduction in IQ,

over a range of Phe from 423 umol/l to 750 umol/l.

-- Significant, proportional correlations were observed between IQ and

mean lifetime blood Phe level for early-treated patients

(r=34; 95% confidence interval), where each 100 umol/l increase in Phe

predicted a 1.9 to 4.1 point reduction in IQ, over a range of Phe from

394 umol/l to 666 umol/l.

-- These statistically significant correlations sugge
'/>"/>

SOURCE BioMarin Pharmaceutical Inc.
Copyright©2007 PR Newswire.
All rights reserved

Page: 1 2 3 4

Related medicine news :

1. Recommendations for Treatment of Blood Pressue
2. Blood Cells Capable of Regenerating Liver
3. Infant receives first bloodless liver transplant
4. Spouses and other partners lower blood pressure says new research
5. H2O - a cure for low blood pressure
6. Uncontrolled blood pressure
7. Weight and Blood Pressure
8. Grandmother’s diet in pregnancy can affect grandchildren’s blood sugr
9. Early diagnosis for autism from blood tests may be possible
10. Antibiotic from crab blood indentified
11. Gum disease may be caused by blood clotting gene
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:7/29/2015)... ... , ... For the second year in a row, the management and staff ... fastest growing companies in Jacksonville. This annual list is compiled by the Jacksonville Business ... of Vast Bridges staffers were on hand to receive the award naming the company ...
(Date:7/29/2015)... ... 2015 , ... Fastline Publications, Inc., will be featured in ... Fastline is dedicated to quality agricultural advertising, both in print and online. In ... searchable agriculture equipment database in the world. The site has equipment listings worth ...
(Date:7/29/2015)... Mobile, AL (PRWEB) , ... July 29, 2015 , ... ... The directory will include the general contact information, website, types of plans administered, years ... submitted their business listing and have at least one plan with Employee Fiduciary. A ...
(Date:7/29/2015)... MO (PRWEB) , ... July 29, 2015 , ... ... of a decision from the Judicial Panel on Multidistrict Litigation (JPML), according to ... birth defects research at their website, the Zofran Birth Defects Lawsuit Center. , ...
(Date:7/29/2015)... ... July 29, 2015 , ... ... a global provider of life insurance, annuities, employee benefits and asset management, has ... inclusive sporting event taking place in Charlotte, North Carolina on October 2-4, ...
Breaking Medicine News(10 mins):Health News:Vast Bridges Continues Its Swift Growth 2Health News:Fastline Publications to Partner with American Farmer in Upcoming Episode Airing on RFD-TV 2Health News:Employee Fiduciary Launches Financial Advisor Directory 2Health News:Zofran Lawsuit News: Zofran Lawsuits Stayed, Awaiting MDL Decision 2Health News:Zofran Lawsuit News: Zofran Lawsuits Stayed, Awaiting MDL Decision 3Health News:Zofran Lawsuit News: Zofran Lawsuits Stayed, Awaiting MDL Decision 4Health News:MetLife Joins World T.E.A.M. Sports’ Adventure Team Challenge North Carolina as Presenting Sponsor 2Health News:MetLife Joins World T.E.A.M. Sports’ Adventure Team Challenge North Carolina as Presenting Sponsor 3
... contracted Lassa fever in Nigeria is being treated at ... Health said on Wednesday. ,"The patient is ... hospital's special isolation ward," said departmental spokesperson, Vusi Sibiya. ... Dr Abdul Rahman, the patient was flown from Abuja ...
... neglected tropical diseases should be given a higher priority ... ,Many people in Southeast Asia suffer from ... yaws. Dengue is emerging as another important problem in ... and ill-health and have considerable economic impact on populations ...
... omega-3 fatty acids may cut the risk of colorectal cancer ... in men not taking// aspirin, suggests new research. ... the food and supplements industry by storm. Much of its ... largely on evidence that it can aid cognitive function and ...
... stanol esters and beta-glucan reduced levels of LDL-cholesterol by ... the Netherlands. ,But whether this could offer ... armoury against elevated cholesterol levels remains to be seen ... possibly due to detrimental interactions between the ingredients. ...
... The African Malaria Network Trust (AMANET) has received a ... & Melinda Gates// Foundation. ,According to the ... the four-year grant is aimed at strengthening institutional capacity ... ,It also aims at fostering and promoting discussion, ...
... colorectal cancers getting missed out in patients undergoing colonoscopy ... gastroenterologist// or a surgeon. This was disclosed by a ... ,This study was conducted on 12,487 colorectal ... along with her team. According to this study, new ...
Cached Medicine News:Health News:South Africa: Nigerian Man Treated for Lassa Fever 2Health News:WHO Urges Higher Priority for Neglected Diseases 2Health News:Omega-3 Again Linked to Lower Colorectal Cancer Risk 2Health News:Combined Ingredients may Cut Cholesterol Levels 2Health News:Combined Ingredients may Cut Cholesterol Levels 3
(Date:7/28/2015)... 28, 2015   Heska Corporation  (NASDAQ:  HSKA -  ... of  advanced veterinary diagnostic and other specialty veterinary ... new Element i ® Immunodiagnostic Analyzer. Element ... key health concerns, including thyroid disorders, Cushing,s disease ... compact bench top analyzer that delivers rapid immunoassay ...
(Date:7/28/2015)... -- Psychemedics Corporation (NASDAQ: PMD ) today announced second ... 2015.  The Company also announced a quarterly dividend of ... of August 7, 2015 to be paid on August ... consecutive quarterly dividend. The Company,s revenue ... million versus $7.7 million for the quarter ended June ...
(Date:7/28/2015)... PARK, Calif. , July 28, 2015 /PRNewswire/ ... medical device company that is providing innovative evidence-based ... announced the publication of the SENZA-RCT results in ... official Journal of the American Society of Anesthesiologists. ... a ground-breaking study that was the first to ...
Breaking Medicine Technology:Heska Announces Veterinary Market's first and only point of care Analyzer for TSH, Total T4, and Cortisol results in minutes 2Heska Announces Veterinary Market's first and only point of care Analyzer for TSH, Total T4, and Cortisol results in minutes 3Psychemedics Corporation Announces Second Quarter Earnings 2Psychemedics Corporation Announces Second Quarter Earnings 3Psychemedics Corporation Announces Second Quarter Earnings 4Psychemedics Corporation Announces Second Quarter Earnings 5Psychemedics Corporation Announces Second Quarter Earnings 6Psychemedics Corporation Announces Second Quarter Earnings 7Psychemedics Corporation Announces Second Quarter Earnings 8Nevro Announces Publication of SENZA-RCT Results in Anesthesiology 2
... ISPE announced today that it has brought ... pharmaceutical industry leaders to be plenary speakers at the ... Baltimore, Maryland, USA on 4 – 5 June 2012. ... the key CGMP issues affecting the pharmaceutical industry today.  ...
... Pharmaceuticals, Inc. (PINKSHEETS: GMPM.PK) a marketing and product ... everyday health and wellness, announced this afternoon, the ... for the Company,s 17-gram Gamma Energy GEL.  The ... Gamma will support sales with point of purchase ...
Cached Medicine Technology:Global Regulatory and Pharmaceutical Industry Leaders to Appear as Plenary Speakers at Joint FDA-ISPE CGMP Conference 2Gamma Pharmaceuticals, Inc. Announces Distribution Agreement for New York Tri-state Area and Purchase Order for Gamma Energy GEL™ 2Gamma Pharmaceuticals, Inc. Announces Distribution Agreement for New York Tri-state Area and Purchase Order for Gamma Energy GEL™ 3
... reference laboratory and a worldwide leader in ... an extensive test menu of highly complex ... anatomic pathology. Owned by the University of ... half of the nation's university teaching hospitals ...
... national reference laboratory and a worldwide leader ... offers an extensive test menu of highly ... and anatomic pathology. Owned by the University ... than half of the nation's university teaching ...
... Laboratories is a national reference laboratory and ... and development. ARUP offers an extensive test ... tests in clinical and anatomic pathology. Owned ... clients include more than half of the ...
... is a national reference laboratory and a ... development. ARUP offers an extensive test menu ... in clinical and anatomic pathology. Owned by ... include more than half of the nation's ...
Medicine Products: