Navigation Links
ASGCT: Gene Therapy Shows Promise for Patients with Pompe Disease
Date:1/30/2010

New research in gene therapy is bringing doctors and patients closer to a comprehensive treatment for a rare disease portrayed in a new Hollywood film, according to the American Society of Gene & Cell Therapy.

Milwaukee (Vocus) January 30, 2010 -- New research in gene therapy is bringing doctors and patients closer to a comprehensive treatment for a rare disease portrayed in a new Hollywood film, according to the American Society of Gene & Cell Therapy.

“Extraordinary Measures” is based on the true story of John Crowley, a father struggling to find treatment for his children with Pompe disease, a rare, and often fatal, inherited disease that severely weakens the heart and lungs. Currently, the only approved treatment for Pompe is the enzyme-replacement medication Myozyme, which was ultimately developed by Genzyme Corporation.

In a study released this week in Molecular Therapy, ASGCT’s official journal, researchers at the University of Florida reported that gene therapy targeted at the diaphragms of mice with Pompe disease improved the ability of the mice to breathe independently.

This summer, six infants with the disease will receive the treatment. Although the treatment is not a cure, when used in conjunction with other therapies, like the drugs portrayed in the film, it is another step toward improving the lives of patients with Pompe disease.

“While the availability of Myozyme has been a life-saving therapy and a breakthrough for those living with Pompe disease, it must serve as a catalyst for continued innovation,” said Robert Mattaliano, PhD, group vice president of protein research and development at Genzyme. “Gene and cell-based therapies are at the forefront of such endeavors and bring the potential go to beyond replacing a missing enzyme by further improving the muscle repair process in this disease and oth
'/>"/>

Source: PRWeb
Copyright©2010 Vocus, Inc.
All rights reserved

Page: 1 2

Related medicine news :

1. AAPM statement on quality radiation therapy
2. Congress Urged to Take Immediate Action to Ensure Frail Patients Continue to Receive Critical Therapy Services
3. Intensive Insulin Therapy Wont Boost Septic Shock Survival
4. Patients with resectable esophageal adenocarcinoma benefit from neoadjuvant chemoradiotherapy
5. Therapy May Relieve Breast Cancer Surgery Complication
6. ALS Therapy Development Institute Receives $1.6 Million Grant From the Department of Defense for Lou Gehrigs Disease Research
7. MBM ScienceBridge GmbH Negotiates License Agreement Between the Georg-August-University Gottingen, Universitatsmedizin, the University of Regensburg, and the UKs Medical Research Council Technology for a New Therapy of Inflammatory and Immune Disea
8. New hope for therapy in heartburn-related cancer
9. Breast cancer multigene test helping patients avoid chemotherapy
10. Psychotherapy May Help Teen Girls Avoid Obesity
11. After Cochlear Implant, Music Therapy May Aid Speech
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:7/1/2015)... ... July 01, 2015 , ... ... products, is helping families get ready for Independence Day celebrations with new takes ... easy and versatile options are perfect for any summertime get together. , “Celebrating ...
(Date:7/1/2015)... , ... July 01, 2015 , ... ... their big summer sale. Customers purchasing two or more Bunion Booties directly ... , Bunion Bootie temporarily corrects misaligned toes and its ultra thin ...
(Date:7/1/2015)... ... ... wasn’t always a steadfast supporter of plastic surgery. But just as her career in ... terrible car accident years ago left her injured and in need of reconstructive surgery ... if I Do?): Answers to All Your Questions about Plastic Surgery” (published by Archway Publishing), ...
(Date:7/1/2015)... ... 01, 2015 , ... B. E. Smith, the only full-service ... a national chief executive officer recruitment for Citizens Medical Center in ... B. E. Smith has recently placed more than 1,000 healthcare executives into organizations. ...
(Date:7/1/2015)... ... 2015 , ... Who doesn’t want a life filled with thriving positivity and ... between work and life, and positive friendships and relationships? , Dr. Marsha W. ... Wellbeing in Doctors. , With a focus on solving the epidemic of ill-being in ...
Breaking Medicine News(10 mins):Health News:Boar’s Head Helps Craft a Delicious Independence Day 2Health News:Boar’s Head Helps Craft a Delicious Independence Day 3Health News:Bunion Bootie, the #1 Non-Surgical Treatment for Bunion Pain, Announces Their Big Summer Sale 2Health News:Francesca Camp’s New Book Unmasks Plastic Surgery Advice 2Health News:Citizens Medical Center Retains B. E. Smith to Recruit New CEO 2Health News:Citizens Medical Center Retains B. E. Smith to Recruit New CEO 3Health News:Medical Doctor Explains The Healthy Effects of a Positive Outlook in New Book 2
... genetics-based explanation for the causes of anorexia nervosa were ... illness than those given a sociocultural explanation, a University ... This is a potentially important finding, said first author ... of information about the biological and genetic underpinnings of ...
... /Xinhua-PRNewswire/ -- Sundia MediTech,(Shanghai) Company recently announced a welcome ... Zhu and Dr. Shu Gao. Dr. Zhu is appointed ... as the Vice,President in charge of Chemical Synthesis business., ... his college,education in chemistry in Nanjing University in China. ...
... will release its fourth quarter and year-end 2007 financial ... The earnings,release will be available on the PPD Web ... On Thursday, February 7, 2008, at 9 a.m. EST, ... to discuss financial results and the,overall business environment. A ...
... CDC Prepares to Release Alarming New HIV Rates; ... Jan. 10 AIDS Healthcare,Foundation (AHF) today urged ... million of proposed budget cuts in AIDS services,that ... services for,Californians living with HIV/AIDS as he seeks ...
... that Program Integrity will,be Needlessly Compromised in the Interim, ... today applauded the Centers for Medicare and Medicaid,Services (CMS) ... need to be accredited, but expressed concern that it ... be fully implemented. The PMC,however, further commends CMS for ...
... for when antiviral drugs fail , , THURSDAY, Jan. 10 ... human proteins that help HIV propagate, creating hope for ... , Using a technique called RNA interference to screen ... according to the study published online Jan. 10 in ...
Cached Medicine News:Health News:People with anorexia less likely to be blamed when biology, genetics explained 2Health News:People with anorexia less likely to be blamed when biology, genetics explained 3Health News:CRO Company Sundia Welcomes New Members to Its Executive Team 2Health News:PPD Announces Fourth Quarter and Year-End 2007 Earnings Release, Webcast and Conference Call 2Health News:AHF to Schwarzenegger: 'AIDS Cuts Are Deadly' 2Health News:AHF to Schwarzenegger: 'AIDS Cuts Are Deadly' 3Health News:Mandatory Accreditation Delayed Until September 2009 2Health News:New Proteins That Help HIV Grow Identified 2
(Date:7/1/2015)... N.J. , July 1, 2015  Echo ... device company focused on non-invasive continuous glucose monitoring ... its new and completely re-designed corporate website ... intuitive access to Echo,s technology and enhances the ... The new website better communicates to prospective customers ...
(Date:7/1/2015)... HEIGHTS, Utah , July 1, 2015 /PRNewswire/ ... ), a manufacturer and marketer of rehabilitation and ... completed its $4,025,000 private placement financing led by ... strategic private equity investor focused on the healthcare ... to promote organic growth and pursue potential strategic ...
(Date:7/1/2015)... , July 1, 2015 ExeGi Pharma, ... and probiotic treatments, announced today that it has been ... and Non-prescription Health Products Directorate (NNHPD) to sell Visbiome™ ... Visbiome was developed by Professor Claudio De Simone ... extensive clinical investigation. Professor De Simone recently signed an ...
Breaking Medicine Technology:Echo Therapeutics Announces New Website Launch 2Echo Therapeutics Announces New Website Launch 3Dynatronics Announces Completion Of $4,025,000 Private Placement Led By Prettybrook Partners 2Dynatronics Announces Completion Of $4,025,000 Private Placement Led By Prettybrook Partners 3ExeGi Pharma Secures Canadian Approval For Well Studied Probiotic 2ExeGi Pharma Secures Canadian Approval For Well Studied Probiotic 3
... 2011 ImaginAb, Inc. and Eurogentec S.A. announce ... for producing engineered antibody fragments. The two companies ... high-performance Pichia pastoris expression system. Leveraging ... the collaboration will accelerate the development of ImaginAb,s ...
... Jan. 31, 2011 Global Real Estate Consultants has ... a new facility in Fremont. Global ... provide insight and transparency in the Fremont commercial real ... Inc,s 350,000 sq. ft. state-of-the-art manufacturing facility with pilot ...
Cached Medicine Technology:ImaginAb, Inc. and Eurogentec S.A. Collaborate to Produce Antibody Fragments in Pichia 2Boehringer Ingelheim to Purchase Amgen's Fremont (USA) Facility 2
Marquette General Health System Reference Laboratory provides a full range of testing including, Chemistry, Hematology, Coagulation, Urinalysis, Microbiology, Cytology, Anatomic Pathology, Flow Cytom...
Headquartered in Burlington, North Carolina, LabCorp has more than 23,500 employees and offers more than 4,400 clinical tests ranging from routine blood analyses to the most sophisticated molecular d...
the LabOne Healthcare Group markets laboratory testing to physicians, patients, managed care companies, self-insured employers, wellness companies and hospitals....
Mayo Medical Laboratories (MML) is the diagnostic reference laboratory for Mayo Clinic. MML specializes in esoteric laboratory testing for health care organizations throughout the United States and a...
Medicine Products: