Navigation Links
Experimental Treatment Could Fight Muscular Dystrophy
Date:8/25/2009

In some patients, injected molecule appears to restart production of missing protein

TUESDAY, Aug. 25 (HealthDay News) -- Injecting a therapeutic molecule into muscle appears to jump-start the production of a crucial protein that's missing in patients with Duchenne muscular dystrophy, British researchers report.

The treatment so far is only applicable to about 13 percent of people with the debilitating and ultimately fatal disease, but scientists are hopeful that similar molecules might expand the treatment to a wider range of patients.

Duchenne muscular dystrophy affects about one in 3,500 males, and involves a progressive wasting of muscle due to a genetic inability to produce the protein dystrophin, a key component of muscle structure. No treatments are available for the illness, and most of those affected die by age 30.

Recently, molecules called antisense oligonucleotides have shown some promise. These molecules work by "skipping over" portions of the defective gene that would otherwise block dystrophin production.

In their study, published online Aug. 25 in The Lancet Neurology, researchers at the University College London Institute of Child Health selected seven patients for whom a particular oligonucleotide molecule, called AVI-4658, appeared suitable. In these patients, the molecule "skipped" exon 51 -- the portion of the dystrophin-blocking gene that appeared to get in the way of effective dystrophin production.

Injecting the molecule into the muscles of these seven patients resulted in increased dystrophin production in all treated muscles, according to a journal news release. "Intramuscular AVI-4658 was safe and induced the expression of dystrophin locally within treated muscles," the team wrote. "On the basis of these observations, we have initiated a dose-ranging study to assess the safety and efficacy of repeated doses of systemic intravenous AVI-4658."

In a commentary, Annemieke Aartsma-Rus and Gert-Jan van Ommen, from Leiden, the Netherlands, noted that while only about 13 percent of Duchenne muscular dystrophy patients can be expected to be helped by AVI-4658, molecules that skip other exons on the gene could be used, potentially spreading the benefit to more than 70 percent of patients.

More information

Find out more about Duchenne muscular dystrophy at the Muscular Dystrophy Association.



-- E.J. Mundell



SOURCE: The Lancet Neurology, news release, Aug. 25, 2009


'/>"/>
Copyright©2009 ScoutNews,LLC.
All rights reserved  

Related medicine news :

1. Experimental Urine Test Spots Appendicitis
2. Senetek PLC Gains Six Year Extension of Rights from Czech Republic Institute of Experimental Botany
3. Experimental drug shows promise against head and neck cancer
4. Experimental agents may prevent radiation-induced leukemia
5. Experimental insulin-like growth factor receptor inhibitor reduced pancreatic cancer growth
6. Hard to Treat Diseases (HTDS) Releases experimental results in the treatment of (MS) Multiple Sclerosis
7. Experimental Drug May Dampen Severe Asthma
8. Experimental Drug May Prevent Plaque Buildup
9. Experimental Biology 2009 meets April 18-22
10. Experimental Weight-Loss Pill Passes Early Test
11. Experimental Drug Fights Bone Marrow Cancers
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Experimental Treatment Could Fight Muscular Dystrophy 
(Date:6/24/2016)... ... June 24, 2016 , ... Global law firm Greenberg Traurig, P.A. ... The attorneys chosen by their peers for this recognition are considered among the top ... Shareholders received special honors as members of this year’s Legal Elite Hall of Fame: ...
(Date:6/24/2016)... Diego, CA (PRWEB) , ... June 24, 2016 , ... ... with the American Cancer Society and the Road To Recovery® program to drive cancer ... to seniors and other adults to ensure the highest quality of life and ongoing ...
(Date:6/24/2016)... ... 24, 2016 , ... People across the U.S. are sharpening their pencils and ... essay contest in which patients and their families pay tribute to a genetic counselor ... National Society of Genetic Counselors (NSGC) Annual Education Conference (AEC) this September. , ...
(Date:6/24/2016)... , ... June 24, 2016 , ... ... is proud to recognize Dr. Barry M. Weintraub as a prominent plastic surgeon ... beautiful women in the world, and the most handsome men, look naturally attractive. ...
(Date:6/24/2016)... Maryland (PRWEB) , ... June 24, 2016 , ... ... Angels is actively feeding the Frederick area economy by obtaining investment capital for ... over the past 2½ years that have already resulted in more than a ...
Breaking Medicine News(10 mins):
(Date:6/24/2016)...   Pulmatrix, Inc ., (NASDAQ: PULM ... announced today that it was added to the Russell ... comprehensive set of U.S. and global equity indexes on ... milestone for Pulmatrix," said Chief Executive Officer Robert ... progress in developing drugs for crucial unmet medical needs, ...
(Date:6/23/2016)... Research and Markets has announced the addition of ... report to their offering. ... kidney failure, it replaces the function of kidneys by removing ... thus the treatment helps to keep the patient body,s electrolytes ... Increasing number of ESRD patients & substantial healthcare expenditure ...
(Date:6/23/2016)... , June 23, 2016 Roche (SIX: ... 510(k) clearance for its Elecsys BRAHMS PCT (procalcitonin) assay ... sepsis or septic shock. With this clearance, Roche is ... a fully integrated solution for sepsis risk assessment and ... with bacterial infection and PCT levels in blood can ...
Breaking Medicine Technology: