Navigation Links
Edison Pharmaceuticals Announces Positive Results of EPI-743 Phase 2A Leigh Syndrome Clinical Trial
Date:9/12/2012

MOUNTAIN VIEW, Calif., Sept. 13, 2012 /PRNewswire/ -- Edison Pharmaceuticals today announced positive results of a recently completed phase 2A study entitled, "Prospective Open Label Study of EPI-743 in Children with Leigh Syndrome (Subacute Necrotizing Encephalomyelopathy)." The study was conducted at the Ospedale Pediatrico Bambino Gesu– the Vatican's children's hospital, Rome, Italy.

Ten children with seven differing subtypes of Leigh syndrome, ranging in age from 1-13 years, were treated with EPI-743. All ten children exhibited reversal of disease progression as measured by four different disease-relevant metrics. The clinical response was durable over 180 days. No significant safety events were observed.

The results of the clinical trial were published on-line September 10, 2012 in the journal Molecular Genetics and Metabolism.

Findings obtained in this prospective phase 2 controlled study confirm previous clinical results obtained in the United States and Europe.

Leigh syndrome is an inherited lethal, progressive, predominately pediatric, neuromuscular disorder for which there are no approved treatments. Initially described in 1951, the hallmarks of the disease include bilateral necrosis (death) of central nervous system regions responsible for the control of breathing and other neurologic functions. Leigh syndrome belongs to a large family of disorders identified as "mitochondrial disease." The disorders share as a common biochemical feature defects in cellular energy metabolism.

EPI-743 is an orally bioavailable small molecule being developed by Edison Pharmaceuticals for the treatment of Leigh syndrome and other inherited mitochondrial diseases. EPI-743 is a member of the para-benzoquinone class of drugs. It serves as a cofactor for the novel drug target– NADPH quinone oxidase 1 (NQO1). Through a redox-based mechanism, EPI-743 augments endogenous glutathione biosynthesis– essential for the con
'/>"/>

SOURCE Edison Pharmaceuticals
Copyright©2012 PR Newswire.
All rights reserved

Page: 1 2

Related medicine technology :

1. Diabecline Antibiotic Receives Edison Award for Best New Pharmaceutical Product
2. Carolinas HealthCare System and Edison Nation Collaborate to Drive Medical Innovation and Technology
3. EMA Grants Orphan Designation to Edison Pharmaceuticals for EPI-743 for Treatment of Leigh Syndrome
4. Savient Pharmaceuticals to Hold First Quarter 2012 Financial Results Conference Call on Wednesday, May 9, 2012
5. Auxilium Pharmaceuticals to Present at the Deutsche Bank 37th Annual Healthcare Conference
6. Nouveau Life Pharmaceuticals (HRID) Contracts with U.S. Manufacturer to Produce New Female Supplement
7. BioElectronics Survey Indicates a Five-Fold Superior Pain Relief Compared to OTC Pharmaceuticals
8. Icahn Issues Statement Regarding Amylin Pharmaceuticals
9. Almost 50 Percent of the Glaucoma Patient Population Remains Untreated, Signifying a Vast Market for Glaucoma Pharmaceuticals, Finds Frost & Sullivan
10. Omthera Pharmaceuticals Announces Positive Top-Line Results from Phase 3 EVOLVE Study
11. Onyx Pharmaceuticals Announces FDA Advisory Committee to Review Carfilzomib for the Treatment of Patients with Relapsed and Refractory Multiple Myeloma
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:9/22/2014)... ALISO VIEJO, Calif. , Sept. 22, 2014 /PRNewswire/ ... today announced that it is offering to sell $200 ... offering.  J.P. Morgan, Deutsche Bank Securities and BofA Merrill ... offering.  Avanir intends to grant the underwriters a 30-day option ... of the shares to be sold in the offering ...
(Date:9/22/2014)... 22, 2014 The western European automated ... is largely a replacement market with fewer new ... acquisitions and partnerships are redefining competitive landscape in ... service provides details about revenue forecasts, unit shipments, ... across the country. The changing landscape makes it ...
(Date:9/22/2014)... , September 22, 2014 ... announced the addition of the  "Global Remotes ...      (Logo: http://photos.prnewswire.com/prnh/20130307/600769) Remotes/remote ... wirelessly operate electronic gadgets such as smart ... from a short line-of-sight distance. Remote controls ...
Breaking Medicine Technology:Avanir Pharmaceuticals Announces Proposed Public Offering of Common Stock 2Avanir Pharmaceuticals Announces Proposed Public Offering of Common Stock 3Automated Endoscope Reprocessor Market Trends in Western Europe 2Automated Endoscope Reprocessor Market Trends in Western Europe 3Global Remotes Market 2014-2018 2Global Remotes Market 2014-2018 3
... 9 Avinger, Inc., a medical device company ... combat peripheral artery disease, announces the enrollment of ... OcclusioN CrossiNg with thE WildCat CatheTer) clinical trial. ... study intended to evaluate the Wildcat Catheter,s ability ...
... (Nasdaq: PCYC ), a biopharmaceutical company focused ... the treatment of cancer and mediated diseases, today announced ... fiscal quarter and fiscal year ended June 30, 2010 ... 2010.  The company will conduct a conference call and ...
Cached Medicine Technology:First Patient Enrolled in 'CONNECT' Clinical Trial 2Pharmacyclics Announces Date of Fiscal Year End 2010 Financial Results and Conference Call and Presentation at Stifel Nicolaus Healthcare Conference 2
(Date:9/22/2014)... Los Angeles, CA (PRWEB) September 22, 2014 ... the life insurance industry is now easier using the ... adults who are trying to local universal life insurance ... , The adult coverage plans that are easily viewed ... companies that underwrite specific lengths of coverage. The plan ...
(Date:9/22/2014)... Teens and young adults at risk for sexually transmitted ... risky sexual behaviors, according to new recommendations from the ... 20 million new sexually transmitted infections such as gonorrhea ... involve people between 15 and 24 years old, according ... published Sept. 22 in Annals of Internal Medicine ...
(Date:9/22/2014)... All sexually active women should be screened for ... chlamydia and gonorrhea, according to new recommendations from ... The task force noted this recommendation includes older ... and pregnant women. Most women with chlamydia ... result, many infections go undetected. If left untreated, ...
(Date:9/22/2014)... ARBOR, Mich. A surprising new study pulls back ... in health care: differences in payment and income between ... who don,t. , Contrary to perception, the research ... to favor surgeons and other procedure-performing doctors. , The ... simple difference in pay per minute explains why doctors ...
(Date:9/22/2014)... New York, New York (PRWEB) September 22, 2014 ... ) filed against Boston Scientific Corp. continue to ... underway in U.S. District Court, Southern District of ... court documents, a new Docket Control Order was ... addresses a number of matters pertaining to the ...
Breaking Medicine News(10 mins):Health News:Universal Life Insurance Prices for Adults Added to National Insurer Portal Online 2Health News:Behavioral Counseling Urged for Teens, Young Adults at Risk for STDs 2Health News:Behavioral Counseling Urged for Teens, Young Adults at Risk for STDs 3Health News:Screen Women for Chlamydia, Gonorrhea, Experts Say 2Health News:Think the system for paying US doctors is rigged to favor surgeons? Study may surprise you 2Health News:Think the system for paying US doctors is rigged to favor surgeons? Study may surprise you 3Health News:Think the system for paying US doctors is rigged to favor surgeons? Study may surprise you 4Health News:Transvaginal Mesh Lawsuits Move Forward, With Issuance of New Order in Federal Boston Scientific Litigation, Bernstein Liebhard LLP Reports 2Health News:Transvaginal Mesh Lawsuits Move Forward, With Issuance of New Order in Federal Boston Scientific Litigation, Bernstein Liebhard LLP Reports 3Health News:Transvaginal Mesh Lawsuits Move Forward, With Issuance of New Order in Federal Boston Scientific Litigation, Bernstein Liebhard LLP Reports 4
... Nyer Medical Group, Inc., (Nasdaq: NYER ) announced ... notifying it of its failure to maintain a minimum closing bid ... for the prior 30 consecutive business days as required by Nasdaq ... the Company has until March 15, 2010, to regain compliance with ...
... ANGELES, Sept. 21 Former House Majority leader Tom Delay, who injured ... all on the line when he and dance partner Cheryl Burke, a ... network. , , Last week, Delay was ... in Marina del Rey, Calif. headed by Dr. Robert S. Bray, Jr., ...
... , RADNOR, Pa., Sept. 21 ... today announced that its TripProtector(SM)( )insurance plans are now ... to $1 million and medical assistance services that include ... 180 countries. Most trip protection plans on the market ...
... dementia, study found , MONDAY, Sept. 21 (HealthDay News) ... people with mild memory problems will soon develop Alzheimer,s ... older people with mild cognitive impairment, and 76 others ... money management test at the start of the study ...
... Montana city, incidents fell but rose again when restrictions were ... ban smoking in bars, restaurants and other public places witness ... , The research -- which incorporated data from a total ... found at least a 17 percent reduction in heart attacks ...
... , EMERYVILLE, Calif., Sept. 21 Bionovo, Inc. (Nasdaq: ... the company received a letter from NASDAQ stating that, for the ... stock has closed below the minimum $1.00 per share requirement for ... that the company has been provided a period of 180 calendar ...
Cached Medicine News:Health News:Nyer Medical Group, Inc., Nasdaq Notification Letter 2Health News:Injured Tom Delay to Put Foot to Test in Debut of Dancing With the Stars Premiere Tonight on ABC 2Health News:HTH Worldwide Introduces Trip Protection Insurance With Major Medical Benefits 2Health News:Mishandling Money May Foreshadow Alzheimer's 2Health News:Smoking Bans Bring a Drop in Heart Attacks 2Health News:Smoking Bans Bring a Drop in Heart Attacks 3Health News:Bionovo Notified by NASDAQ of Minimum Bid Price Deficiency 2Health News:Bionovo Notified by NASDAQ of Minimum Bid Price Deficiency 3
Hardy pituitary spoon, bayonet, malleable....
... Spinal Implant system. Material that has a ... bone. The result is an implant that ... support, while optimizing the fusion environment through ... anatomic design with 5 and 10 degrees ...
... provides anterior column support for single or ... to L5. The system consists of one ... that are stacked to a desired height, ... Fiber Reinforced Polymer material emulates the biomechanical ...
A new generation of cage suitable for both PLIF and ALIF indications....
Medicine Products: