Navigation Links
Infants With Rare Genetic Disease Saved by Cord Blood Stem Cells

d normally, according to Kurtzberg.

Kurtzberg said their treatment successes provide a strong argument for mandatory, universal screening of all newborns for the genetic lysosomal storage diseases. The major barrier to universal newborn screening is building an infrastructure to support widespread testing, she said. The cost of testing itself would be relatively low.

Although lysosomal storage diseases are rare ?fewer than 5,000 births combined out of 4 million per year in the U.S. ?they are best treated if patients are diagnosed and treated in early infancy, said Kurtzberg. In fact, transplantation in newborns yields the best long-term outcomes and newborns tolerate the transplants more easily than older babies and children.

"Time is of the essence in treating these children before symptoms progress and become irreversible," said Kurtzberg, director of Duke's Pediatric Blood and Marrow Transplant Program. "Delaying the transplant even a week or two can make a noticeable difference in a child's motor skills, so timely diagnosis and treatment is essential to preserving brain and motor function."

Children transplanted after onset of symptoms do stabilize but exhibit minimal neurologic improvement, said Maria Luisa Escolar, MD, of UNC-CH, whose team assessed the neurological, cognitive and physical development of each child with Krabbe Disease before and after transplant.

Escolar is lead author of the study and directs the Program for Neurodevelopmental Function in Rare Disorders at the Center for Development and Learning, UNC-CH. The program is dedicated to tracking the natural history and the effects of new therapies in children with rare diseases.

In the new study, eleven asymptomatic newborns (ages 12 to 44 days) and 14 symptomatic infants (ages 142 days to a year) were treated with unrelated donor umbilical cord blood. Cells from the donor "engrafted" or took hold in all of the patients. All of the asymptomatic newbo
'"/>

Source:Duke University


Page: 1 2 3 4

Related biology news :

1. Infants with Rare Genetic Disease Saved By Cord Blood Stem Cells
2. Antiretroviral Therapy May Prevent HIV Transmission From Breastfeeding Mothers To Infants
3. Infants can organise visual information at just four months
4. Novel Asthma Study Shows Multiple Genetic Input Required; Single-gene Solution Shot Down
5. Genetically modified natural killer immune cells attack, kill leukemia cells
6. Ants Genetic Engineering Leads To Species Interdependency
7. Genetic Variation Visualization - From EMBL
8. Genetically modified rice in China benefits farmers health, study finds
9. Genetically Modified Natural Killer Immune Cells Attack, Kill Leukemia Cells
10. Genetic defects give the immune system the green light to attack the pancreas
11. Maine Researchers Find Exceptions to Old Rules of Genetic Inheritance
Post Your Comments:
*Name:
*Comment:
*Email:
TAG: Infants With Rare Genetic Disease Saved Cord Blood Stem Cells

(Date:7/27/2015)... , July 27, 2015   Zynx Health ... experience-based clinical improvement solutions, today announced that its ... now available on Android smartphones and tablets. With ... post-care organizations can use ZynxCarebook to securely exchange ... condition, streamline care transitions to other care settings, ...
(Date:7/21/2015)... Connecticut , July 21, 2015  NXT-ID, ... "Company"), a biometric authentication company focused on the ... Wocket® smart wallet, announces that it has filed ... System and Method. This invention ... not only authorizes an account, but also the ...
(Date:7/13/2015)... Jul. 13, 2015 NXT-ID, Inc. (NASDAQ: ... authentication company focused on the growing mobile commerce ... patent 62/188684 for BEHAVIORAL-DIRECTED AUTHENTIATION METHOD ... convenient and secure method to make payments.  With ... methods, introduced with its groundbreaking voice-direct payment patent ...
Breaking Biology News(10 mins):Zynx Health Adds Android Device Support to ZynxCarebook Solution 2Zynx Health Adds Android Device Support to ZynxCarebook Solution 3NXT-ID Patents Personalized Tokenized Payments for Next Generation Secure Payment Technology 2NXT-ID Patents Personalized Tokenized Payments for Next Generation Secure Payment Technology 3NXT-ID Patents Personalized Tokenized Payments for Next Generation Secure Payment Technology 4NXT-ID Patents Behavior-Directed Payments 2NXT-ID Patents Behavior-Directed Payments 3NXT-ID Patents Behavior-Directed Payments 4
... to hear relies on hair cells, sensory receptors that mechanically ... transduction channel. Although the transduction channel was characterized more than ... molecular components. A new study in The Journal ... definitive identification of this mystery channel. Recent studies ...
... a long and complex process. Candidate drugs are evaluated ... of animals, usually rats or mice. Each class of ... often in a labor-intensive process that leaves plenty of ... consensus that current procedures fail to effectively produce new ...
... Clinical Practice Guideline (CPG) for the diagnosis and treatment ... in women of reproductive age and a leading cause ... of Polycystic Ovary Syndrome: An Endocrine Society Clinical Practice ... the Journal of Clinical Endocrinology and Metabolism ...
Cached Biology News:Finding psychiatric drugs in the movements of mice 2Finding psychiatric drugs in the movements of mice 3Experts clarify conflicting criteria for diagnosing polycystic ovary syndrome 2
(Date:8/3/2015)... 2015  MiMedx Group, Inc. (NASDAQ: MDXG ), ... and patent-protected processes to develop and market advanced products ... Sports Medicine, Ophthalmic and Dental sectors of healthcare, announced ... Annual Canaccord|Genuity Growth Conference in Boston, ... CEO, William C. Taylor , President and COO, ...
(Date:8/3/2015)... -- Merck KGaA, Darmstadt, Germany ,  a ... the healthcare, life science and performance materials sectors, announced ... and launch of promising new fullerene derivatives for the ... Germany , is pursuing many projects ... Novel derivatives: highly stable, easy to process and cost ...
(Date:8/3/2015)... 2015  Aethlon Medical, Inc. (Nasdaq: AEMD ), ... life-threatening diseases, today announced that it will host its ... on Thursday, August 13, 2015 at 4:30 p.m. Eastern ... the release of fiscal 2016 first quarter results.  A ... two hours after the call at the Investor Relations ...
(Date:8/3/2015)... Aug. 3, 2015   Intrexon Corporation (NYSE: ... half and second quarter 2015 financial results after the ... The Company will host a conference call at 5:30 ... general business update. The conference call may ... (International), and asking to join the "Intrexon Corporation Call."  ...
Breaking Biology Technology:MiMedx To Present At 35th Annual Canaccord|Genuity Growth Conference 2Merck KGaA, Darmstadt, Germany, and Nano-C Present Enhanced Materials for Organic Photovoltaics 2Merck KGaA, Darmstadt, Germany, and Nano-C Present Enhanced Materials for Organic Photovoltaics 3Merck KGaA, Darmstadt, Germany, and Nano-C Present Enhanced Materials for Organic Photovoltaics 4Aethlon Medical To Host First Quarter Fiscal 2016 Investor Call on August 13, 2015 2Intrexon to Announce First Half and Second Quarter 2015 Financial Results and Host Conference Call on August 10th 2
... 2011 Reportlinker.com announces that a ... in its catalogue: What,s ... and Other Biologics) 2011-2015 ... future of the pharmaceutical market will ...
... aid the fight of the deadly outbreak of E. ... at the University Medical Centre Hamburg-Eppendorf have released an ... data previously released and submitted to NCBI in the ... assembled using the latest ion semiconductor sequencing technology, there ...
... Asmacure Ltee, a clinical-stage biopharmaceutical company focused ... treatment of asthma and other inflammatory diseases, announced today ... officer.  With a solid foundation established at Asmacure, Mr. ... lead compound, a potential new treatment for asthma with ...
Cached Biology Technology:Reportlinker Adds What's Next in Biologics? (mAbs, rDNA, Interferons, and Other Biologics) 2011-2015 2Reportlinker Adds What's Next in Biologics? (mAbs, rDNA, Interferons, and Other Biologics) 2011-2015 3Reportlinker Adds What's Next in Biologics? (mAbs, rDNA, Interferons, and Other Biologics) 2011-2015 4Reportlinker Adds What's Next in Biologics? (mAbs, rDNA, Interferons, and Other Biologics) 2011-2015 5Reportlinker Adds What's Next in Biologics? (mAbs, rDNA, Interferons, and Other Biologics) 2011-2015 6Reportlinker Adds What's Next in Biologics? (mAbs, rDNA, Interferons, and Other Biologics) 2011-2015 7Further Analysis on Improved Genome Assembly Indicates the Outbreak E. coli has Complex Genetics With Resistance to at Least Eight Antibiotics 2Further Analysis on Improved Genome Assembly Indicates the Outbreak E. coli has Complex Genetics With Resistance to at Least Eight Antibiotics 3Asmacure Ltee Appoints Pharmaceutical and Biotechnology Industry Veteran Martin Driscoll as CEO 2Asmacure Ltee Appoints Pharmaceutical and Biotechnology Industry Veteran Martin Driscoll as CEO 3
Rabbit polyclonal to Glutathione S Transferase alpha ( Abpromise for all tested applications). Antigen: Full length protein: Human Glutathione S Transferase alpha. Entrez GeneID: 2938 Swi...
Affinity Purified Rabbit Anti-mouse sRANKL Biotinylated Polyclonal Antibody...
Mouse anti-IkBa/MAD-3 Class: Antibody Product Group: Signalling molecules and phospho-specific Antibody...
Mouse monoclonal [002-49] to C1s ( Abpromise for all tested applications). entrezGeneID: 716 SwissProtID: P09871...
Biology Products: