Navigation Links
Drug fights cystic fibrosis
Date:2/5/2008

BIRMINGHAM, Ala. An experimental drug that has proven effective in treating muscular dystrophy also works for cystic fibrosis, according to researchers at the University of Alabama at Birmingham (UAB).

The new study is the latest on a compound called PTC124, which helps to rescue faulty proteins that lead to illnesses. The drug holds promise in treating more than 2,400 genetic diseases caused by a certain class of DNA mutation.

In the UAB tests performed on mice, PTC124 restored to normal function up to 29 percent of the cases of abnormal cystic-fibrosis (CF) protein.

The findings are published in the online version of the journal Proceedings of the National Academy of Sciences and will soon appear in a print edition.

The study adds to research published last year in the journal Nature, which showed PTC124 restored up to 25 percent of the missing or abnormal protein function in mice with Duchenne muscular dystrophy.

Our study shows strong pre-clinical evidence that PTC124 is capable of suppressing nonsense mutations that cause cystic fibrosis, said David Bedwell, Ph.D., professor in the UAB Department of Microbiology and lead author on the study. We think this provides strong evidence for clinical trials with PTC124 in CF patients with this kind of mutation.

A gene that carries a nonsense mutation produces a shortened or faulty protein that degrades in the body. The absence of that protein is what leads to disease, Bedwell said. An estimated one-third of gene defects responsible for human disease are thought to come from nonsense mutations. In the case of CF, the absence of a certain protein leads to an imbalance of salt and water in the linings of the lungs and other membranes.

The UAB study showed that PTC124 allowed the protein to be made in mouse cells where it was previously absent, and it was delivered in a specific location that helped restore salt and water balance in membranes.


'/>"/>
Contact: Troy Goodman
tdgoodman@uab.edu
205-934-8938
University of Alabama at Birmingham
Source:Eurekalert

Page: 1 2

Related medicine news :

1. MIT radar technology fights breast cancer
2. New Drug Fights Medication-Linked Bone Loss
3. Experimental Drug Fights Prostate Cancer
4. Connecticut Girl Fights Rare Neurological Disease
5. Two-Drug Combo Fights Brain Tumors
6. Guacamole: Fights Hunger, Cholesterol and Cancer
7. Secondhand Smoke Worsens Lung Function in Cystic Fibrosis Patients
8. Cystic Fibrosis Foundation reports upward trend for key health outcomes
9. Montana Passes Newborn Screening Law for Cystic Fibrosis
10. Ibuprofen Preserves Lung Function in Kids With Cystic Fibrosis
11. Ibuprofen associated with slower lung function decline in children with cystic fibrosis
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:8/27/2014)... Los Angeles, CA (PRWEB) August 27, 2014 ... everyone. To do this, Jaha invested in creating the industry’s ... motivation by adding functions that no other apps possess. ... with a tracker that differentiates in three simple steps: locate, ... will launch on Tuesday, Aug 26, 2014 to garner support ...
(Date:8/27/2014)... The Global and China Maleic Anhydride ... current state of the Maleic Anhydride industry in Global ... of the industry, including definitions, applications and industry chain ... are provided with a focus on history, developments, trends ... the international and Chinese situation is also offered. Get ...
(Date:8/27/2014)... New Haven, CT (PRWEB) August 27, 2014 ... , CRN International is about to shed some light on ... exclusive research to members of the Brand Activation Association ... The 40-year-old marketing company polled hundreds of consumers to learn ... have the biggest impact on influencing a potential buying decision. ...
(Date:8/27/2014)... 2014 BCC Research ( http://www.bccresearch.com ) ... IMPLANTS AND REGENERATIVE PRODUCTS , the global market for ... billion by 2014-end to $42.2 billion by 2019, with ... 2014 to 2019. New technologies and an increasingly older ... the market. , While there are many conventional and ...
(Date:8/27/2014)... HealthDay Reporter TUESDAY, Aug. 26, 2014 ... for blood transfusions might also protect the brains of premature ... stimulates red blood cell production, appears to prevent brain damage ... Petra Huppi, a professor of pediatrics and newborn medicine at ... of whether EPO protects the brains of these children will ...
Breaking Medicine News(10 mins):Health News:Jaha, Inc. The Most Affordable Fitness Band in the Market Released on Kickstarter 2Health News:Maleic Anhydride Industry & Isoprene Market Global and China Analysis Now Available at ReportsnReports.com 2Health News:Maleic Anhydride Industry & Isoprene Market Global and China Analysis Now Available at ReportsnReports.com 3Health News:Maleic Anhydride Industry & Isoprene Market Global and China Analysis Now Available at ReportsnReports.com 4Health News:Maleic Anhydride Industry & Isoprene Market Global and China Analysis Now Available at ReportsnReports.com 5Health News:CRN Set To Release Poll Results On What Marketing Tactics Prompt Consumers To Buy 2Health News:Global Market for Advanced Orthopedic Technology to Reach $42.2 Billion in 2019; Biotechnological Advancements Driving the Market 2Health News:Global Market for Advanced Orthopedic Technology to Reach $42.2 Billion in 2019; Biotechnological Advancements Driving the Market 3Health News:Hormone Might Help Preemies' Brains 2Health News:Hormone Might Help Preemies' Brains 3
... ARBOR, Mich.---Persons with alcohol problems are finding comfort in ... shows. Among 1,910 people with any alcohol-related problems, ... researchers at the University of Michigan Health System and ... who used services from clergy also used professional services ...
... Today during the 88th General Session & Exhibition ... Spain, presenting author J. Meyle, Justus Liebig University, Giessen, ... Infection and Immune Evasion of Oral Carcinomas." ... oral cancers and periodontal disease. They achieved results by ...
... By Steven Reinberg HealthDay Reporter , TUESDAY, ... long shadow. Those who survive the original cancer are ... new cancers, heart disease and stroke likely caused by ... more children are surviving cancer, many have long-term risks ...
... associated with cancer symptoms often unrecognized and ... telephone-based care management coupled with automated symptom monitoring, ... Indiana University School of Medicine. The Indiana ... calls with follow-up calls from the nurse care ...
... A report issued by the Program on International Health ... Health proposes a new research agenda to address the ... "The Pregnancy Intentions of HIV-Positive Women: Forwarding the ... urges a multi-disciplinary research approach to help HIV-positive women ...
... By Amanda Gardner HealthDay Reporter , TUESDAY, ... the safety of controversial diabetes drug Avandia, members of the ... skeptical of much of the data presented. A decision ... The hearing has coincided with news that the drug,s ...
Cached Medicine News:Health News:Individuals confess alcohol abuse to clergy 2Health News:Childhood Cancer Survivors at Risk of Premature Death 2Health News:Childhood Cancer Survivors at Risk of Premature Death 3Health News:Help is on the phone: Reducing pain and depression of cancer 2Health News:Report proposes new research agenda on pregnancy intentions of HIV-positive women 2Health News:Report proposes new research agenda on pregnancy intentions of HIV-positive women 3Health News:FDA Panel Seems Skeptical Over Key Avandia Data 2Health News:FDA Panel Seems Skeptical Over Key Avandia Data 3Health News:FDA Panel Seems Skeptical Over Key Avandia Data 4
(Date:8/27/2014)... , Aug. 27, 2014 IRIDEX Corporation (NASDAQ: ... James H. Mackaness is scheduled to make an investor ... at 2:55 p.m. Eastern Time on Wednesday, September 3, in ... of the investor presentation will be available in the Investors ... About IRIDEX IRIDEX Corporation was founded ...
(Date:8/27/2014)...   Auxilium Pharmaceuticals , Inc. (NASDAQ: ... announced that certain members of the executive management team ... be held September 8-10, 2014 in New ... Officer and President, is scheduled to present an overview ... a.m. ET on Wednesday, September 10, 2014. ...
(Date:8/27/2014)... Calif. , Aug. 27, 2014 Asterias ... U.S. Food and Drug Administration (FDA) to initiate a ... patients with complete cervical spinal cord injury.  The approved ... clinical study of the product, and is designed to ... in patients with complete cervical spinal cord injuries, the ...
Breaking Medicine Technology:Auxilium Pharmaceuticals, Inc. To Present At The Morgan Stanley Healthcare Conference 2Auxilium Pharmaceuticals, Inc. To Present At The Morgan Stanley Healthcare Conference 3Asterias Biotherapeutics Receives U.S. FDA Clearance to Initiate Phase 1/2a Clinical Trial of AST-OPC1 in Patients with Cervical Complete Spinal Cord Injury 2Asterias Biotherapeutics Receives U.S. FDA Clearance to Initiate Phase 1/2a Clinical Trial of AST-OPC1 in Patients with Cervical Complete Spinal Cord Injury 3Asterias Biotherapeutics Receives U.S. FDA Clearance to Initiate Phase 1/2a Clinical Trial of AST-OPC1 in Patients with Cervical Complete Spinal Cord Injury 4Asterias Biotherapeutics Receives U.S. FDA Clearance to Initiate Phase 1/2a Clinical Trial of AST-OPC1 in Patients with Cervical Complete Spinal Cord Injury 5
... Highlights from Avanafil Development Program Include: ... trials Comparative in-vitro selectivity of avanafil, sildenafil, ... post-prostatectomy population VIVUS, Inc. (NASDAQ: ... presented at the Sexual Medicine Society of North America (SMSNA) ...
... Campden Publishing Ltd today launched its medical and educational ... with the launch, Campden Health Communications has separately announced ... that enables its readers to upload video content directly ... magazine image. This is the first time the ,Aurasma, ...
Cached Medicine Technology:Avanafil Data to be Presented at the Sexual Medicine Society of North America Meeting 2Avanafil Data to be Presented at the Sexual Medicine Society of North America Meeting 3Avanafil Data to be Presented at the Sexual Medicine Society of North America Meeting 4Avanafil Data to be Presented at the Sexual Medicine Society of North America Meeting 5Campden Publishing Unveils new Med Comms Division and World-First Technology Licence 2
Instrument Cover for Vibratome....
These specialty razor blades are feather thin and very sharp. Most users have found an increased quality level in Vibratome sections with these blades....
This is a perfect tool for retrieving free floating sections from the Vibratome bath....
The care package includes a spare motor, fuse, Spare Fluorescent Bulb, additional Vibratome Feather Blades, Specimen Adhesive, Specimen Mounting Blocks, and a Specimen Tray....
Medicine Products: