Navigation Links
Making treatment of rare blood disorder more affordable and effective
Date:2/27/2014

PHILADELPHIA A University of Pennsylvania research team has defined a possible new way to fight a disease that is currently treatable only with the most expensive drug available for sale in the United States. In a study published this month in Blood, the Penn team describes the strategy, based on the oldest part of the human immune system called "complement" -- that could turn out to be less costly and more effective for the majority of patients with a rare blood disorder.

Complement is a network of more than 50 proteins in the blood and on cell surfaces that quietly cruise the body, keeping a low profile until triggered into action. On the other hand, this system can turn, contributing to a broad spectrum of immune, inflammatory, and age-related diseases.

John Lambris, PhD, the Dr. Ralph and Sallie Weaver Professor of Research Medicine in the Department of Pathology and Laboratory Medicine in the Perelman School of Medicine, studies this early-warning system and how to correct it when its response goes overboard.

This is known to be the case with paroxysmal nocturnal hemoglobinuria (PNH), a rare but life-threatening hematological disorder, which is estimated to affect between 1 and 5 per million people. In PNH, defective expression of regulatory proteins on the surface of blood cells leaves them vulnerable to complement attack. This can lead to premature death of the red blood cells, a process called hemolysis, which clinically results in severe anemia and contributes to a high risk of clotting.

Lambris and Daniel Ricklin, PhD, research assistant professor of Pathology and Laboratory Medicine, are developing novel therapeutics to tame this inappropriate complement activation and protect cell surfaces from attack.

Eculizumab (Soliris, an Alexion Pharmaceuticals drug) -- to date the only approved therapeutic for PNH -- reduces hemolysis and gives relief from blood transfusions for most PNH patients. However,
'/>"/>

Contact: Karen Kreeger
karen.kreeger@uphs.upenn.edu
215-349-5658
University of Pennsylvania School of Medicine
Source:Eurekalert

Page: 1 2

Related medicine news :

1. Brain Changes May Hamper Decision-Making in Old Age
2. OHSU Oregon National Primate Research Center develops new, safer method for making vaccines
3. After 25 years, World No Tobacco Day is making an impact
4. Making bad worse for expectant mothers
5. DotComSecrets’ Russell Brunson Is Revealing Online Money Making Secrets in Free Webinars for Students
6. New Electronic Cigarette Free Trial Kit Adds More Years to Smokers Life by Making it Easy to Quit Smoking
7. First Is Viewed as Best When Making Quick Decisions
8. Neuroeconomics to study decision-making in anxious individuals
9. Making it easier to learn to read
10. The making and unmaking of stem-like, aggressive breast cancer cells
11. UH students spent summer making a difference with hands-on research
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:7/25/2014)... CA (PRWEB) July 25, 2014 AttorneyOne.com, a ... the latest information from the FDA on Ibuprofen and ... 21, that one lot of Ibuprofen and one lot of ... due to mislabeled packaging. Oxcarbazepine is used for the ... , The reason for the recall is that ...
(Date:7/25/2014)... 2014 A new study of participants in ... are published today in Cancer , found evidence that ... who had an abnormal finding on a lung cancer screening ... life than did those who were screened and found to ... to the researchers. “We expected that participants with an abnormal ...
(Date:7/25/2014)... TX (PRWEB) July 25, 2014 ... the current state of the Galactooligosaccharides (GOS) ... a basic overview of the industry, including definitions, ... and Chinese domestic market analysis are provided with ... landscape of the market. A comparison between the ...
(Date:7/25/2014)... July 24, 2014 (HealthDay News) -- Shift workers, especially men, ... to people not on such schedules, a new study suggests. ... work on a set schedule, with shifts moving around at ... at all surprising," said one expert, Dr. Alan Manevitz, a ... "Physicians have long known that working shifts disrupts many ...
(Date:7/25/2014)... -- A new system that sent patients to designated ... victims of sudden cardiac arrest in Arizona, according to ... of Emergency Medicine . , "We knew lives ... cutting edge guidelines for post-cardiac arrest care and we ... hospitals, similar to what is done for Level 1 ...
Breaking Medicine News(10 mins):Health News:Ibuprofen and Oxcarbazepine Tablets Recalled: AttorneyOne Monitors and Keep Consumers Informed 2Health News:Full Explanation Reduces Seniors’ Worries about Lung Cancer Screening 2Health News:Full Explanation Reduces Seniors’ Worries about Lung Cancer Screening 3Health News:Full Explanation Reduces Seniors’ Worries about Lung Cancer Screening 4Health News:Full Explanation Reduces Seniors’ Worries about Lung Cancer Screening 5Health News:GOS Market (Galacto-oligosaccharides) Development in Global & China Regions Now Available at DeepResearchReports.com 2Health News:GOS Market (Galacto-oligosaccharides) Development in Global & China Regions Now Available at DeepResearchReports.com 3Health News:Study Links Shift Work to Risk for Type 2 Diabetes 2Health News:Study Links Shift Work to Risk for Type 2 Diabetes 3Health News:New EMS system in Arizona dramatically improves survival from cardiac arrest 2Health News:New EMS system in Arizona dramatically improves survival from cardiac arrest 3
... People with a mild fear of flying should take ... their breath for six to 10 seconds, just before ... Professional Association of German Psychiatrists (known by its German ... takes the mind off one's fears. ,BVDP ...
... order to prevent discrimination against the people suffering from ... on potential employees. // ,Satyam is the ... test on candidates selected for jobs in its various ... we have stopped conducting the HIV/AIDS test to protect ...
... provide free care to the poor has dropped over ... problem for America’s quickly growing number of uninsured people. ... the Center for Studying Health System Change, released on ... the mid-1990s, compared with about two-thirds now. This is ...
... down. The industry across the country is now facing a suicide ... the country, small farmers are scared due to low prices. // ... Rs 5 for one kg of chicken from Rs 1.80 and ... being forced to commit suicide. K Narayana Reddy, president, of Andhra ...
... offer a pay of nearly $500,000 to the head of ... proposal, the Queensland Opposition has described it as being extravagant ... advertised for the post of a new director for the ... $500,000-plus. This is believed to pave way for enormous ...
... person who had popularized country music in the United States ... died on // Saturday. He was 76., ,Owens' family said ... according to the website of a concert hall and restaurant he ... Aug 12, 1929, in Texas to a poor farming family, Alvis ...
Cached Medicine News:Health News:Satyam Stops Conducting HIV Tests On Employees To Prevent Discrimination 2Health News:Fewer Doctors Treating For Free 2
(Date:7/25/2014)... Cardinal Health will today name three of the nation,s ... Independent Pharmacy Best Practices Competition, during the company,s ... gatherings of independent pharmacists. The Best ... Ohio , Tennessee and ... implementing exceptionally creative methods that demonstrate how independent pharmacies ...
(Date:7/25/2014)... N.Y. and MEXICO CITY , July 25, 2014 ... that PLM, a Latin American healthcare sciences publisher, launched ... medical information on conditions such as cancer and heart ... access the most up-to-date information to deliver better patient ... is growing rapidly, with many relying on smartphones and ...
(Date:7/25/2014)...  Semler Scientific, Inc. (Nasdaq: SMLR ... that develops patented products that assist healthcare providers ... reported financial results for the second quarter and ... "The second quarter of 2014 continued to ... , M.D., chief executive officer of Semler.  "We ...
Breaking Medicine Technology:Cardinal Health Announces Three Best Practice Winners for Leading Change in Community Pharmacy 2Cardinal Health Announces Three Best Practice Winners for Leading Change in Community Pharmacy 3Cardinal Health Announces Three Best Practice Winners for Leading Change in Community Pharmacy 4Cardinal Health Announces Three Best Practice Winners for Leading Change in Community Pharmacy 5Cardinal Health Announces Three Best Practice Winners for Leading Change in Community Pharmacy 6Healthcare Sciences Publisher PLM and IBM Speed Access to Medical Information to Deliver Better Patient Care with Mobile 2Healthcare Sciences Publisher PLM and IBM Speed Access to Medical Information to Deliver Better Patient Care with Mobile 3Healthcare Sciences Publisher PLM and IBM Speed Access to Medical Information to Deliver Better Patient Care with Mobile 4Semler Reports Second Quarter 2014 Financial Results 2Semler Reports Second Quarter 2014 Financial Results 3
... Oct. 29 Ferring Pharmaceuticals Inc. announces the appointment ... for Orthopaedics. Dr. Kovelman will be responsible for ... treatment for knee pain due to osteoarthritis, as well ... Kovelman is exceptionally well qualified to fill this role," ...
... NANJING, China, Oct. 29 /PRNewswire-Asia/ -- Simcere Pharmaceutical ... a leading pharmaceutical company,specializing in the development, manufacturing, ... China, today announced that it has,entered into an ... a Cayman,Islands company that, as its sole business, ...
Cached Medicine Technology:Dr. Harry Kovelman Joins Ferring Pharmaceuticals as Associate Medical Director for Orthopaedics 2Dr. Harry Kovelman Joins Ferring Pharmaceuticals as Associate Medical Director for Orthopaedics 3Simcere Pharmaceutical Group to Acquire Additional Stake in Jiangsu Yanshen Biological Technology Stock Co., Ltd. 2Simcere Pharmaceutical Group to Acquire Additional Stake in Jiangsu Yanshen Biological Technology Stock Co., Ltd. 3
... practices performing routine Holter,monitoring, the ... Wizard provides a,practical, no-nonsense diagnostic ... health care,environment. This highly accurate ... a variety,of configurations at a ...
... Burdick EK-10 single-channel,electrocardiograph features ... membrane keypad. It provides ... a quick and,accurate diagnosis. ... with a built in ...
The CSV-1000ETDRS10% test face presents the standard ETDRS test at a contrast level of 10%. This test is widely used in clinical studies for the evaluation of refractive surgery and contact lenses....
... test face is used widely for screening ... LogMAR acuity from 20/10 to 20/100 and ... cycles/degree. , ,The patient can be easily ... acuity and contrast sensitivity. If a contrast ...
Medicine Products: