Navigation Links
An 'elegant' idea proves its worth 25 years later

The simple notion of copying the body’s own natural "waste disposal" chemistry to mop up potentially toxic nitrogen has saved an estimated 80 percent of patients with urea cycle disorders --- most of them children – according to a report in this week’s New England Journal of Medicine summarizing a quarter century of experience with the treatment.

The effectiveness of sodium phenylacetate and sodium benzoate, two chemicals the body already makes to carry nitrogen for disposal in urine "just knocked my socks off from the moment we first tried them," recalls Saul Brusilow, M.D., professor emeritus of pediatrics at Hopkins who first had the notion to use the drugs. "In all my years I never came across another disease where patients come in near-comatose and you stick a needle in them and lo and behold, they wake up—just like that. It was just astonishing," he says.

"His elegant idea was to give patients chemicals they already make in small amounts in large doses to make up for the missing urea cycle enzyme they inherited," says Ada Hamosh, M.D., M.P.H, clinical director of the McKusick-Nathans Institute of Genetic Medicine. "Sodium phenylacetate and sodium benzoate already know how to eliminate nitrogen in urine, so having more in the body carries more nitrogen out and reduces the toxic effects of excess nitrogen accumulation."

Excess nitrogen yields ammonia, which is poisonous and in the case of urea cycle disorders, causes brain damage, retardation, coma and even death.

Despite the immediate clinical success of the treatment, the drug combination was finally approved by the U.S. Food and Drug Administration only in 2005.

Brusilow, Hamosh, and colleagues at Stanford University, University of Minnesota, Thomas Jefferson University and the Medical College of Wisconsin looked back at 299 urea cycle disorder patients with a total of 1,181 hyperammonemia "episodes" from 118 hospitals around the United States from August 1
'"/>

Source:Johns Hopkins Medical Institutions


Page: 1 2

Related biology news :

1. Nobel Laureate finds elegant explanation for DNA transcribing enzymes high fidelity
2. Adding Radiation Therapy To Chemotherapy Improves Survival In Patients With High-risk Breast Cancer
3. FDA Approves Human Hookworm Vaccine for Phase I Safety Trials
4. Purdue proves concept of using nano-materials for drug discovery
5. FDA Approves New Drug to Treat Type I and Type II Diabetes
6. Canada approves marijuana-based pain spray
7. Bevacizumab Combined With Chemotherapy Improves Progression-Free Survival for Patients With Advanced Breast Cancer
8. Product improves peptide identification for proteomics research
9. FDA Approves New Treatment for Chronic Hepatitis B
10. Stem cell treatment improves mobility after spinal cord injury
11. FDA approves more generic AIDS drugs
Post Your Comments:
*Name:
*Comment:
*Email:
TAG: elegant idea proves its worth years later

(Date:8/20/2015)... PLEASANTON, Calif. , Aug. 20, 2015 /PRNewswire/ ... focused on consumers that are active and healthy. ... used in the hospital environment to help improve ... released a video from the Wearable Technologies ... MD discussed new applications for wearables in healthcare.    ...
(Date:8/18/2015)... PRINCETON, N.J. , Aug. 18, 2015 ... be a key contributor to the Submission ... hosted by IRISS, a non-profit forum. Dedicated to ... IRISS provides a neutral platform for industry, ... to share information and work towards a ...
(Date:8/12/2015)... As the digital payment services ... advanced biometrics technology is accelerating beyond expectations.  While the ... using their credit cards, the digital currency, mobile payment ... services led by companies in the space such as ... (NASDAQ: GOOG ), Apple Inc. (NASDAQ: AAPL ...
Breaking Biology News(10 mins):Leaf Healthcare Releases Video from Wearable Technology Conference on New Applications for Wearables in the Hospital Environment 2Leaf Healthcare Releases Video from Wearable Technology Conference on New Applications for Wearables in the Hospital Environment 3Navitas, a Key Contributor in IRISS Forum Webinar 2Navitas, a Key Contributor in IRISS Forum Webinar 3Digital Wallet Devices & Mobile Payment Management Services Gain a Distinct Advantage in the Digital Payment Evolution as Demand Continues to Grow 2Digital Wallet Devices & Mobile Payment Management Services Gain a Distinct Advantage in the Digital Payment Evolution as Demand Continues to Grow 3Digital Wallet Devices & Mobile Payment Management Services Gain a Distinct Advantage in the Digital Payment Evolution as Demand Continues to Grow 4Digital Wallet Devices & Mobile Payment Management Services Gain a Distinct Advantage in the Digital Payment Evolution as Demand Continues to Grow 5Digital Wallet Devices & Mobile Payment Management Services Gain a Distinct Advantage in the Digital Payment Evolution as Demand Continues to Grow 6
... have a mixed reputation. Their delicate, colorful flowers and ... gardeners and growers. But the striking plants are often ... warm temperatures, high humidity, and fertilizer is required to ... recently published study by scientists Julia C. Brotton and ...
... tree native to North America, is native from northern ... the United States, where it grows abundantly along the ... and Texas. Popularity and consumer demand for pecans has ... while commercial production has expanded into many regions of ...
... (Mon., Nov. 2, 2009) -- The introduction of high-throughput ... the genetics of complex diseases. Instead of focusing only ... sample of individuals, the ability to accurately and efficiently genotype ... individual genes has resulted in genetic studies with greater statistical ...
Cached Biology News:For African violets, 'hands off' means healthier 2Sun or shade: Pecan leaves' photosynthetic light response evaluated 2High-throughput genotyping, protein purification featured in Cold Spring Harbor Protocols 2
(Date:8/27/2015)... - BIOREM Inc. (TSXV: BRM) ("Biorem" or "the Company") today announced ... 2015. Biorem,s complete second quarter financial statements and MD&A have been ... except earnings per shareThree-months ended , Six -months endedJune 30 , ... , 2014 Revenue , 4,709 , ... , 1,656 , 56 , 3,188 ...
(Date:8/27/2015)... -- GlassesOff (OTCBB: GLSO), a visual neuroscience technology ... vision system, announced today a partnership with eight-time All-Star, ... to develop a new mobile app designed for professional ... on-court performance. Vision is the inception of every ... ball or blocking a pass. The critical impact vision speed ...
(Date:8/27/2015)... ... August 27, 2015 , ... ... and expertly narrows down the product choices to a manageable shortlist of bench ... the list to models that fit best with the customer’s industry and application. ...
(Date:8/27/2015)... ... August 27, 2015 , ... Proove Biosciences, ... data on the relationship of genetics and hypertension at the International Academy ... in Vancouver, British Columbia, Canada. The event, which boasts an extensive overview of ...
Breaking Biology Technology:BIOREM Reports $718,000 Net Earnings for the Second Quarter 2BIOREM Reports $718,000 Net Earnings for the Second Quarter 3Eight-Time All-Star Chris Paul and GlassesOff Partner to Develop Innovative Mobile App Designed to Improve On-Court Performance 2Eight-Time All-Star Chris Paul and GlassesOff Partner to Develop Innovative Mobile App Designed to Improve On-Court Performance 3METTLER TOLEDO Updates Online Scale Selection Tool 2Proove Biosciences Presents Ground-Breaking Data on Genetics and Heart Disease at 20th World Congress on Heart Disease 2Proove Biosciences Presents Ground-Breaking Data on Genetics and Heart Disease at 20th World Congress on Heart Disease 3
... Laboratories,( http://www.microtestlabs.com ) has doubled their microbial ... an additional MicroSeq(R),Microbial Identification System., The ... that,enables Microtest technicians to more quickly and ... or easily identified., "With the purchase ...
... 3 Associates of Cape Cod,Inc. (ACC), a ... announced that the Fungitell(R) assay kit, for the,measurement ... CE marked, which enables distribution in the European ... included in both the Infectious,Disease Society of America,s ...
... iCAD, Inc. (Nasdaq:,ICAD), an industry-leading provider of ... Chief Executive Officer, and,Darlene Deptula-Hicks, Executive Vice President ... a corporate update at the Seventh Annual,Needham and ... 11, 2008 at 2:00 p.m., (Eastern Daylight Time) ...
Cached Biology Technology:Microtest Labs Doubles Microbial Identification, Analytical Services 2Fungitell(R) CE Marked, Referenced in IDSA and NCCN Guidelines and Added to EORTC-MSG Revised Definitions of Invasive Fungal Disease 2Fungitell(R) CE Marked, Referenced in IDSA and NCCN Guidelines and Added to EORTC-MSG Revised Definitions of Invasive Fungal Disease 3iCAD to Present at Seventh Annual Needham and Company Biotechnology and Medical Technology Conference 2
...
... ( Abpromise for all tested ... Synthetic peptide: IGPEVPDDRDFEPS conjugated to KLH, ... Human Decorin (fragment 36-49 of the ... Entrez GeneID: 1634 ...
...
... all Programmable and Digital Orbital Shaker. Our durable, ... to make every day use even easier. This ... up to 4 different programs. Its easy recall ... Furthermore, the VSOS-4P has a pulse operation for ...
Biology Products: