Navigation Links
First Gene Therapy Human Trial For Muscular Dystrophy In Progress

ao told Samulski he wanted to put his new minidystrophin gene into an AAV vector and test it.

"He wanted to know if UNC could make the virus, and that's when I told him that we were using this virus for Canavan's disease and for other efforts. We had started improving the vectors, and we had developed some new ones that we thought were better for muscle."

Xiao and Samulski put their projects together and formed Asklepios BioPharmaceutical Inc. (AskBio) in 2003. Along with the rights granted by UNC to Samulski's vector technology, AskBio acquired the intellectual property rights to Xiao's uniquely miniaturized dystrophin gene.

In July 2004, the Muscular Dystrophy Association (MDA) awarded $1.6 million to AskBio to develop gene therapy strategies for DMD.

Following extensive laboratory toxicity experiments required by the U.S. Food and Drug Administration demonstrating that minidystrophin gene transfer was unlikely to harm and could ultimately benefit muscles affected by DMD, approval was granted March 3, 2006, to proceed with the human trial.

"After years of encouraging pre-clinical results, I'm excited that AskBio will help bring this promising new therapy into the clinic, and look forward with a great deal of optimism to offering this initial step toward hope for the DMD community," Samulski said.

--Eurekalert
'"/>




Page: 1 2 3

Related medicine news :

1. First Vaccine Designed for Africa Cleared for Testing in Humans
2. Ajanta Launches Worlds First Once-A-Day Nimesulide Oral Formulation
3. First human clone is near
4. First Artificial Heart patient has Major setback
5. First head-to-head trials of once weekly Fosamax and Actonel therapies
6. WHO Declares Vietnam First Country to Control SARS
7. Russia Reports First SARS Case
8. Hope for First New Melanoma Treatment in Decades
9. First global SARS meet opens
10. Stanford Medical Center Implements The First Virtual Cardiac Ultrasound
11. Hyderabad To Have Asia’s First Catheter Making Uni
Post Your Comments:
*Name:
*Comment:
*Email:
TAG: First Gene Therapy Human Trial For Muscular Dystrophy Progress

(Date:7/25/2014)... Ticket Down announces that they ... Guinness International Champions Cup match between international soccer powerhouses ... on Sunday, July 27th. This well-known ticket ... SOCCER-2014 for added savings. , 2014 is turning out ... in North America. Countless international soccer teams have been ...
(Date:7/25/2014)... (GSA) the nation,s largest interdisciplinary organization ... chosen Heather M. Young, PhD, RN, FAAN, of ... recipient of the Doris Schwartz Gerontological Nursing Research ... Health Sciences Section, is given to a member ... sustained contribution to gerontological nursing research. , The ...
(Date:7/25/2014)... Island (PRWEB) July 25, 2014 Reproductive ... to announce that Dr. Kerri Luzzo, Lead Physician at ... Award" for her professional accomplishments in fertility care and ... , At the 10th Annual PBN Awards ceremony held ... Island, 40 of the most highly regarded, emerging leaders ...
(Date:7/25/2014)... The Little Gym of Houston will ... July 27 at Discovery Green in downtown Houston. The ... Pearland, Friendswood, Spring, The Woodlands, Sugarland-Missouri City, Kingwood, Bellaire ... “We’re thrilled to be attending and performing at the ... owns the Sugarland-Missouri City location. “We believe in the ...
(Date:7/25/2014)... (PRWEB) July 25, 2014 AttorneyOne.com, a recognized ... latest information from the FDA on Ibuprofen and Oxcarbazepine ... that one lot of Ibuprofen and one lot of Oxcarbazepine ... due to mislabeled packaging. Oxcarbazepine is used for the treatment ... , The reason for the recall is that Lot ...
Breaking Medicine News(10 mins):Health News:Liverpool FC vs. Olympiacos FC Tickets Soldier Field: Ticket Down Slashes Ticket Prices for Guinness International Champions Cup in Chicago on Sunday, July 27th 2Health News:Young earns GSA's 2014 Doris Schwartz Gerontological Nursing Research Award 2Health News:Dr. Kerri Luzzo of the Reproductive Science Center of Providence Receives “40 Under Forty Award" for Outstanding Professional Accomplishments and Community Outreach 2Health News:The Little Gym of Houston to Attend Houston Family Magazine’s Family Fun Fest 2014 2Health News:Ibuprofen and Oxcarbazepine Tablets Recalled: AttorneyOne Monitors and Keep Consumers Informed 2
... Texas, hospital go online with electronic health recordsNASHVILLE, ... (HMS) announced today that they and Cogdell Memorial ... Memorial selected HMS to supply a fully integrated ... the Snyder, Texas hospital closer to electronic health ...
... Jan. 7 The TV Teacher, LLC(TM) announces the,donation ... York City schools: Queens, Bronx, and Staten Island. , ... Beats(TM) was co-created,by Susan Ellis, a mother struggling to ... the alphabet. After an unsuccessful two years of,"traditional" ...
... Snow Jr., chairman and chief executive officer, Medco Health Solutions, ... the 27th Annual JPMorgan Healthcare Conference in San Francisco on ... Hotel, San Francisco, will begin at 8:30 a.m. PST (11:30 ... Richard J. Rubino, chief financial officer, and Robert S. Epstein, ...
... Mass., Jan. 7 Boston Scientific Corporation (NYSE: ... upcoming JP Morgan Health Care Conference in San Francisco.Jim ... on Wednesday, January 14th, beginning at approximately 12:00 p.m. ... President and Chief Financial Officer will join Mr. Tobin ...
... Trademark Office (USPTO) has registered Guru Program® as ... developed and honed by Tom Gable, of Gable ... USPTO language said the mark covered: consulting services ... legal and accounting services, health care, financial services, ...
... 7 Agito Networks, Inc.(TM), the,company delivering ... today,announced that Technology Marketing Corporation,s (TMC(R)) Unified,Communications ... Networks,RoamAnywhere(TM) Mobility Router(TM) as a recipient of ... Agito Networks, award-winning RoamAnywhere ...
Cached Medicine News:Health News:Cogdell Memorial Hospital Partners with Healthcare Management Systems 2Health News:Cogdell Memorial Hospital Partners with Healthcare Management Systems 3Health News:New York Schools Receive Program for Children with Autism 2Health News:Medco CEO to Present at the 27th Annual JPMorgan Healthcare Conference 2Health News:Guru Program Pioneered by Tom Gable Receives Official Registration from USPTO 2Health News:Guru Program Pioneered by Tom Gable Receives Official Registration from USPTO 3Health News:Agito Networks' RoamAnywhere Mobility Router Wins Unified Communications Magazine's 2008 UC Excellence Award 2Health News:Agito Networks' RoamAnywhere Mobility Router Wins Unified Communications Magazine's 2008 UC Excellence Award 3Health News:Agito Networks' RoamAnywhere Mobility Router Wins Unified Communications Magazine's 2008 UC Excellence Award 4
(Date:7/25/2014)... PORTLAND, Ore. , July 25, 2014 /PRNewswire/ ... "Semler"), an emerging medical risk assessment company that ... monitoring patients and evaluating chronic diseases, today reported ... months ended June 30, 2014. ... productive for Semler," said Doug Murphy-Chutorian , ...
(Date:7/25/2014)... /PRNewswire-iReach/ -- Dr. Michael Gabriel of a prominent ... a recent study, which shows how grief in pregnancy may ... According to US News , ... likely than others to grow up overweight or obese, even ... were two specific types of stress that caused obesity: "Children ...
(Date:7/24/2014)... MADISON, Wis. , July 24, 2014 /PRNewswire/ ... provides specialty and compounded pharmaceuticals, announced that it ... sensitivity information unique to each individual based upon ... now possible by understanding the interaction between a ... "We,re very excited to provide this ...
Breaking Medicine Technology:Semler Reports Second Quarter 2014 Financial Results 2Semler Reports Second Quarter 2014 Financial Results 3Dr. Michael Gabriel of GPM Pediatrics, a Noted Staten Island Pediatrics Clinic, Comments on Study, Which Names Grief as a Cause of Obesity in Children 2Restore Health Launches Pharmacogenetic Testing, Enhancing Its Suite of Personalized Medicine Goods and Services 2Restore Health Launches Pharmacogenetic Testing, Enhancing Its Suite of Personalized Medicine Goods and Services 3
... 10 Bayer HealthCare announced today the recipients ... Nursing Fellowship (IHNF).  This global initiative, supported by Bayer, ... countries – areas where hemophilia-trained healthcare professionals are in ... countries ( Algeria , Brazil , Cameroon ...
... July 9 Pioneer Surgical Technology, Inc. recently expanded its Biologics ... their growing line of products that address bone and soft tissue repair. ... Mr. Shane ... with our ability to provide innovative biologic products and to compete with ...
Cached Medicine Technology:Bayer HealthCare Announces Recipients of 2010 WFH International Hemophilia Nursing Fellowship 2Bayer HealthCare Announces Recipients of 2010 WFH International Hemophilia Nursing Fellowship 3Bayer HealthCare Announces Recipients of 2010 WFH International Hemophilia Nursing Fellowship 4Pioneer® Surgical Expands Product Specialists to Support Growing Biologics Business 2
... be used in facilities where space is ... ventilation and gas delivery technologies with an ... and the functionality of the Draeger Infinity ... yourself a workplace environment that is a ...
... superior ventilation., ,The Aestiva/5 offers exceptional ... approach to anesthesia therapy., ,The Aestiva/5 ... an intensive care ventilator, saving you the ... into the OR. Its flexible ergonomic design ...
With its integrated design and superior build quality each Prima SP is custom built to meet your requirements....
A non-adhering hydrogel sheet dressing for use on all partial and full thickness wounds (excluding 3rd degree burns.) Hydrogel sheet contains 96% water and 4% insoluble cross-linked polyethylene oxid...
Medicine Products: