Navigation Links
Clinical Trial Shows Some Promise for Duchenne Muscular Dystrophy Drug
Date:7/18/2017

The results of an international clinical trial, led in part by a UC Davis School of Medicine expert, have shown that a drug therapy for a severe type of muscular dystrophy holds promise for a subgroup of patients.

In a paper just published online in the journal The Lancet, Craig McDonald and colleagues at 53 study sites in 18 countries describe the clinical benefit of using the drug ataluren for a certain group of patients carrying a specific “nonsense mutation” for Duchenne muscular dystrophy.

Duchenne muscular dystrophy is a rare but fatal genetic disorder characterized by progressive muscle degeneration and weakness, which is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. The so-called nonsense mutation, a genetic mutation in the DNA sequence that results in a shorter, non-functional protein, is present in about 13 percent of Duchenne patients and makes the disease more severe.

McDonald and his co-authors say treatments focusing on dystrophin restoration, such as ataluren, are important because they can preserve existing muscle function, which otherwise progressively deteriorates.

“What’s very encouraging about these findings is that a treatment using ataluren for certain individuals with Duchenne muscular dystrophy may prompt a slowing or stabilizing of the disease progression and motor function that can be very meaningful to patients and provide them with invaluable, quality-of-life benefits,” said McDonald, who was the lead author on the study and serves as professor and chair of the UC Davis Department of Physical Medicine and Rehabilitation.

The study, a randomized, double-blind Phase 3 clinical trial, was sponsored by the New Jersey-based company that developed ataluren, PTC Therapeutics. It included 230 patients, half of whom received the drug therapy over a course of 48 weeks, and the other half who received a placebo.

To assess patient muscle function and mobility as well as drug efficacy, researchers used a six-minute walk test, which was developed by McDonald and his team at UC Davis. Duchenne study participants with the nonsense mutation who had a baseline six-minute walk of between 300 meters and under 400 meters were the subgroup of patients who were the most likely to see the clinical benefits of ataluren.

“While not all of the ataluren-treated participants reached statistical significance, this subgroup seems to represent a stage in the progression of disease where dystrophin restoration therapy, as measured by the six-minute walk test, does have value,” added McDonald, who has been a consultant to drug manufacturing companies working on therapies for Duchenne, including PTC Therapeutics. “Our findings certainly merit additional study because the patients and families I see in clinic every week are desperate for help.”

Duchenne muscular dystrophy primarily affects males. The lack of functional dystrophin protein affects the structural stability of skeletal, diaphragm and heart muscles. Patients with the disease lose the ability to walk as early as age 10 and experience life-threatening lung and heart complications in their late teens and twenties.

Of the approximately 250,000 people with the disorder worldwide, the nonsense mutation in Duchenne accounts for about 13 percent of cases, which represents approximately 2,000 patients in the U.S.

Read the full story at http://www.prweb.com/releases/2017/07/prweb14519391.htm.


'/>"/>
Source: PRWeb
Copyright©2017 Vocus, Inc.
All rights reserved


Related medicine news :

1. Kidney Week 2013: New innovations in clinical science
2. Salar Joins Medical Informatics Experts to Judge AMIA’s First Ever Student Design Challenge: Reinventing Clinical Documentation
3. The DelRay Recovery Center Appoints Britt Bonner as the Director of Clinical Outreach
4. Clinical trial looks at impact of platelet-rich plasma therapy on tennis elbow
5. Nemours receives Dept. of Defense award to study physicians maintenance of optimal clinical program
6. Clinical trial finds concurrent therapy not necessary to achieve high pathological in breast cancer
7. Catalent’s Clinical Trial Supply Facility in Shanghai, China Opens for Business
8. Italian study examines clinical predictors of acute urinary symptoms after radiotherapy for prostate
9. Idol Lash: The Clinically Proven Eyelash Growth Enhancer Now Available in the Market With 1 Extra Bottle Offer
10. Paid Asthma Clinical Trial Now Enrolling at Achieve Clinical Research Near Birmingham, Alabama; Accepting M/F Patients with Asthma Age 12 - 75
11. Tinnitus Global Clinical Trials Review, H2, 2013
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:9/21/2017)... ... September 21, 2017 , ... The American ... Health Services for professionals in the addiction treatment industry entitled: Special Investigations ... , Insurance companies and state and federal governments are increasingly scrutinizing the addiction ...
(Date:9/21/2017)... ... 21, 2017 , ... Hair restoration surgery is having a ... the International Society of Hair Restoration Surgery (ISHRS). The worldwide volume ... with 635,189 procedures performed in 2016. , "Like other forms of cosmetic ...
(Date:9/21/2017)... , ... September 21, 2017 , ... Bill Howe Plumbing’s ... its customers, and give back to the community. For over 37 years, they have ... successful companies serving plumbing in San Diego. They were chosen as the ...
(Date:9/21/2017)... ... September 21, 2017 , ... Innovatum ... at the Global GS1 Healthcare Conference 2017 in Chicago, IL on October 17-19, ... ensuring label quality and improving patient safety. , Microscan , a ...
(Date:9/21/2017)... ... September 21, 2017 , ... ... and highly accurate cancer screening tests, has received two prestigious recognitions that acknowledge ... survival rates. , Preora has been named a Top 100 Finalist ...
Breaking Medicine News(10 mins):
(Date:9/9/2017)... , Sept. 8, 2017 Dealmed Medical ... independent supplier of medical equipment, supplies, drugs, vaccines, and ... has entered into an agreement to acquire Vantage Medical ... based in Holtsville, New York . ... in supplying new and emerging medical practices, will operate ...
(Date:9/7/2017)... Sept. 7, 2017 NuvoAir (formerly called ... announced today a partnership with Novartis Pharma AG to distribute ... position as the leading mobile spirometry platform and Novartis, commitment ... ... ...
(Date:9/6/2017)...   PDI , a leader in infection prevention ... educational session focused on the role of chlorhexidine gluconate ... the 2017 Annual Scientific Meeting of the Association for ... the Phoenix Convention Center in ... will also feature PDI,s Prevantics® Device Swab ...
Breaking Medicine Technology: