Navigation Links
Experimental Drug Offers Hope for Rare Bone Disease: Study
Date:3/7/2012

By Serena Gordon
HealthDay Reporter

WEDNESDAY, March 7 (HealthDay News) -- A new therapy may be the first to offer hope for children born with a rare disease that affects bone development, sometimes so severely that babies die because they're missing a rib cage to protect their lungs.

The inherited disorder is called hypophosphatasia, and the new medication is asfotase alfa. It works by replacing an enzyme that's missing in those with hypophosphatasia. Enzymes are substances responsible for speeding up certain chemical reactions. In hypophosphatasia, the missing enzyme is necessary for proper bone growth and normal metabolism.

A small study of babies and children younger than 3 who had debilitating or life-threatening hypophosphatasia found that treatment with asfotase alfa strengthened bones and improved lung function. After 48 weeks of treatment, many could start bearing weight on their legs and some infants were even taking their first steps.

"We saw striking improvements in these patients with severe hypophosphatasia who received the enzyme replacement," said the study's lead author, Dr. Michael Whyte, medical-scientific director of the Center for Metabolic Bone Disease and Molecular Research at Shriners Hospitals for Children in St. Louis. Whyte is also a professor at Washington University School of Medicine in St. Louis, which conducted the study jointly with Shriners and other institutions.

Results of the research are published in the March 8 issue of the New England Journal of Medicine.

Severe hypophosphatasia affects about 1 in 100,000 babies born in the United States, according to the National Library of Medicine. It's estimated that more people may have the disease, but in far milder forms. The severity of the disease can range from life-threatening to simply causing dental problems in adults, according to background information in the article.

The enzyme in hypophosphatas
'/>"/>

Copyright©2010 ScoutNews,LLC.
All rights reserved  

Page: 1 2 3

Related medicine news :

1. Experimental Drug Offers Hope for Cystic Fibrosis Patients
2. Experimental Vaccine Shields Monkeys Against Ebola
3. Experimental targeted therapy shows early promise against medulloblastomas
4. Experimental nonsteroidal treatment of asthma shows promise
5. Experimental obesity drug avoids brain effects that troubled predecessors
6. Experimental treatments for cocaine addiction may prevent relapse
7. Experimental TB Test Called Fast and Accurate
8. Experimental Drug Shows Promise for Bone Marrow Disorder
9. Experimental Leukemia Drug Proves a Slam Dunk
10. Experimental Test May Spot Prostate Cancer Earlier, More Accurately
11. Experimental Drug Aids Kids With Nervous System Tumor
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Experimental Drug Offers Hope for Rare Bone Disease: Study
(Date:8/27/2015)... ... August 27, 2015 , ... Thriveology Founders, Dr. Carly Letzt Carney and ... Amazing Aging Expo in Denver, Colorado on Saturday, August 29, 2015 at the Marriott ... media outlets as health authorities, and will present a two part seminar entitled, “How ...
(Date:8/27/2015)... ... August 27, 2015 , ... On May 30th, 2015, at the ... as sponsor of the “Music With A Mission” benefit concert. For the past two ... programs in the underfunded school districts of Mendon and neighboring town, Quincy, IL. Organized ...
(Date:8/27/2015)... , ... August 27, 2015 , ... The Naderi Center ... be offering Kybella, a cutting-edge, noninvasive procedure to improve submental fat. The first ... provides permanent results without surgery. Dr. Kulak is the only Board Certified surgeon ...
(Date:8/27/2015)... CA (PRWEB) , ... August 27, 2015 , ... Healthpointe ... into its La Tijera Clinic in Los Angeles. Licensed to practice Chinese Needle Therapy ... emphasis on neuromuscular injuries and diseases. Dr. Priebe also practices in Healthpointe’s La Mirada ...
(Date:8/27/2015)... (PRWEB) , ... August 27, 2015 , ... In addition ... Alvernia University now offers a third doctoral program at its main campus in Reading: ... a terminal degree program designed to prepare advanced practice nurses for the highest level ...
Breaking Medicine News(10 mins):Health News:Internationally-renowned Naturopathic Doctor and Chiropractor Team up to Reveal Secrets of Avoiding Disease 2Health News:Best Drug Rehabilitation Interviews Madd Hoss Jackson at the 2015 "Music With A Mission" Benefit Concert 2Health News:Noninvasive Double Chin Elimination Now Available in Herndon and Chevy Chase 2Health News:Noninvasive Double Chin Elimination Now Available in Herndon and Chevy Chase 3Health News:Qualified Medical Examiner and Acupuncturist Joins Healthpointe in Los Angeles 2Health News:Qualified Medical Examiner and Acupuncturist Joins Healthpointe in Los Angeles 3Health News:Alvernia University Now Offering Three Doctoral Degree Programs 2
... ... Center published in Brain Injury, the official journal of the International Brain Injury Association, ... (TBI). Cognitive fatigue has been shown to be one of the most challenging symptoms ... ...
... , DENVER, June 11 Birner Dental Management Services, ... of PERFECT TEETH dental practices, announced its regular ... a quarterly cash dividend of 17 cents per share of ... to shareholders of record June 26, 2009. , , ...
... external defibrillators , , WASHINGTON, June 11 In ... (SCA), The Richard B. and Lynne V. Cheney Cardiovascular Institute ... ReStart DC program on June 11. The goal of the ... where large numbers of people gather throughout all eight wards ...
... to Stem Growing Healthcare Coverage Shortfalls - , , WASHINGTON, ... 1221, "The Medicare Prompt Pay Correction Act," a companion ... of Representatives and currently has 45 co-sponsors. , , ... Pat Roberts (R-KS). The bill is a step forward in addressing ...
... His Life , , DALLAS, June 11 In his ... but later years and the pressure of being a father and ... depression Bresee created the poetry of his book, "The Hidden Story: ... ). , , "Depression and addictions brought me to a ...
... /PRNewswire-FirstCall/ - MDS Inc. (TSX: MDS; NYSE: MDZ ... global life sciences markets, today reported financial results for the ... MDS reported total revenue of $282 million, a net loss ... for the second quarter of 2009. These results include a ...
Cached Medicine News:Health News:Kessler Foundation Research Center Study Provides Insight into One of the Most Challenging Symptoms Following a Traumatic Brain Injury 2Health News:Kessler Foundation Research Center Study Provides Insight into One of the Most Challenging Symptoms Following a Traumatic Brain Injury 3Health News:ReStart DC Launches Life-Saving Public Access Defibrillation Program 2Health News:ReStart DC Launches Life-Saving Public Access Defibrillation Program 3Health News:Senate Bill to Protect Patients' Healthcare by Amending Medicare Coverage 2Health News:Senate Bill to Protect Patients' Healthcare by Amending Medicare Coverage 3Health News:'The Hidden Story': Poetry Inspired by Author's Struggle with Depression, Addiction 2Health News:MDS Reports Second Quarter 2009 Financial Results 2Health News:MDS Reports Second Quarter 2009 Financial Results 3Health News:MDS Reports Second Quarter 2009 Financial Results 4Health News:MDS Reports Second Quarter 2009 Financial Results 5Health News:MDS Reports Second Quarter 2009 Financial Results 6Health News:MDS Reports Second Quarter 2009 Financial Results 7Health News:MDS Reports Second Quarter 2009 Financial Results 8Health News:MDS Reports Second Quarter 2009 Financial Results 9Health News:MDS Reports Second Quarter 2009 Financial Results 10Health News:MDS Reports Second Quarter 2009 Financial Results 11Health News:MDS Reports Second Quarter 2009 Financial Results 12Health News:MDS Reports Second Quarter 2009 Financial Results 13Health News:MDS Reports Second Quarter 2009 Financial Results 14Health News:MDS Reports Second Quarter 2009 Financial Results 15Health News:MDS Reports Second Quarter 2009 Financial Results 16Health News:MDS Reports Second Quarter 2009 Financial Results 17Health News:MDS Reports Second Quarter 2009 Financial Results 18Health News:MDS Reports Second Quarter 2009 Financial Results 19
(Date:8/27/2015)... 27, 2015   NineSigma the leading innovation partner ... in estimated overall revenue growth and anticipates the creation of ... Open Innovation Incentive (OII). In 2013, the ... grant of over $2 million to accelerate adoption of open ... Million and $1 Billion in revenues. To date over 45 ...
(Date:8/27/2015)... 27, 2015  Hill-Rom Holdings, Inc., (NYSE: HRC ... at the Morgan Stanley Global Healthcare Conference on September ... are invited to listen to the live discussion via ... directly at https://cc.talkpoint.com/morg007/091615a_ae/?entity=49_UCJPU8N . A recorded ... after the conclusion of the live event and accessible ...
(Date:8/27/2015)... TORONTO , Aug. 27, 2015  Generex Biotechnology ... issuance of a white paper on the status of ... The white paper was presented to the August 19, ... James H. Anderson, Jr. , MD, the Company,s ... online on the Generex website. The white ...
Breaking Medicine Technology:State of Ohio and NineSigma Collaborate to Drive Regional Economic Growth and Job Creation 2State of Ohio and NineSigma Collaborate to Drive Regional Economic Growth and Job Creation 3Generex Announces Issuance of Buccal Insulin White Paper 2Generex Announces Issuance of Buccal Insulin White Paper 3Generex Announces Issuance of Buccal Insulin White Paper 4
... Reportlinker.com announces that a new market research ... Global Kidney Stone Management ... http://www.reportlinker.com/p0611035/Global-Kidney-Stone-Management-Devices-Industry.html#utm_source=prnewswire&utm_medium=pr&utm_campaign=Medical_D This report analyzes ... Devices in US$ Million by the following ...
... RMD ) announced today that it will be presenting ... Conference at the Grand Hyatt in New York City on September ... Grand Hyatt in New York City on September 20, 2011; ... in Phoenix, AZ on November 9, 2011; Piper Jaffray Healthcare ...
Cached Medicine Technology:Global Kidney Stone Management Devices Industry 2Global Kidney Stone Management Devices Industry 3Global Kidney Stone Management Devices Industry 4Global Kidney Stone Management Devices Industry 5Global Kidney Stone Management Devices Industry 6Global Kidney Stone Management Devices Industry 7Global Kidney Stone Management Devices Industry 8Global Kidney Stone Management Devices Industry 9Global Kidney Stone Management Devices Industry 10Global Kidney Stone Management Devices Industry 11ResMed Inc. to Present at Upcoming Conferences 2
... The Zeus Scientific, Inc. ENA Profile-6 ... for the detection of IgG antibodies to ... in human sera. When performed according to ... autoantibody profile may aid in the diagnosis ...
Inquire...
... K-ASSAY Microalbumin Urine Controls are ... use liquid requiring no reconstitution ... from human urine, fortified to ... and creatinine. Preservatives including sodium ...
The K-ASSAY ITA Lyophilized Multi-Control Set is intended to be used as a consistent test sample of known concentration for monitoring the performance of K-ASSAY immunoturbidimetric assays for the li...
Medicine Products: