New muscular dystrophy drug's chances for approval improve

April 22, 2014
New muscular dystrophy drug's chances for approval improve
A new drug to treat Duchenne muscular dystrophy may be closer to becoming the first approved treatment for the disease.

(HealthDay)—A new drug to treat Duchenne muscular dystrophy may be closer to becoming the first approved treatment for the disease.

Eteplirsen was created to treat Duchenne muscular dystrophy, which affects about one in 3,500 boys worldwide. The drug seemed to be on track for swift approval from the U.S. Food and Drug Administration, but that effort stalled late last year after a similar type of for the disease failed in a clinical trial, the Washington Post reported.

However, Massachusetts-based Sarepta Therapeutics said Monday that FDA officials outlined a potential way forward for the drug and said they may consider it for accelerated approval.

"This provides the opportunity to get the drug approved and in the hands of all the boys who can benefit from it sometime in 2015," Sarepta chief executive officer Chris Garabedian told the Post.

Explore further: New drug improves walking performance for Duchenne muscular dystrophy patients

Related Stories

New drug improves walking performance for Duchenne muscular dystrophy patients

August 8, 2013
Results from a clinical trial of eteplirsen, a drug designed to treat Duchenne muscular dystrophy, suggest that the therapy allows participants to walk farther than people treated with placebo and dramatically increases production ...

Docs: Heart device might be breakthrough for muscular dystrophy

October 17, 2012
(HealthDay)—A man with Duchenne muscular dystrophy who received a device to help his heart's left ventricle pump blood throughout his body could represent a breakthrough in the treatment of the disease, according to his ...

Treating Duchenne muscular dystrophy

December 2, 2013
(Medical Xpress)—Reviving a gene which is 'turned down' after birth could be the key to treating Duchenne muscular dystrophy (DMD), an incurable muscle-wasting condition that affects one in every 3,500 boys.

Regenerating muscle in Duchenne muscular dystrophy: Age matters

April 14, 2014
A team of scientists led by Pier Lorenzo Puri, M.D., associate professor at Sanford-Burnham Medical Research Institute (Sanford-Burnham), in collaboration with Fondazione Santa Lucia in Rome, Italy, have published details ...

New breakthrough could help treat muscular dystrophy

August 13, 2012
A researcher in the Faculty of Medicine & Dentistry at the University of Alberta improved Duchenne muscular dystrophy symptoms in non-human lab models, using a new drug cocktail. The drug combination targets the “hot ...

Discovery one step closer to treatment for Duchenne muscular dystrophy

July 30, 2013
(Medical Xpress)—A University of Alberta researcher has pinpointed a mutation that brings the medical community another step closer to treating those who suffer from a fatal type of muscular dystrophy. 

Recommended for you

In most surgery patients, length of opioid prescription, number of refills spell highest risk for misuse

January 17, 2018
The possible link between physicians' opioid prescription patterns and subsequent abuse has occupied the attention of a nation in the throes of an opioid crisis looking for ways to stem what experts have dubbed an epidemic. ...

Patients receive most opioids at the doctor's office, not the ER

January 16, 2018
Around the country, state legislatures and hospitals have tightened emergency room prescribing guidelines for opioids to curb the addiction epidemic, but a new USC study shows that approach diverts attention from the main ...

FDA bans use of opioid-containing cough meds by kids

January 12, 2018
(HealthDay)—Trying to put a dent in the ongoing opioid addiction crisis, the U.S. Food and Drug Administration on Thursday slapped strict new restrictions on the use of opioid-containing cold and cough products by kids.

Taking ibuprofen for long periods found to alter human testicular physiology

January 9, 2018
A team of researchers from Denmark and France has found that taking regular doses of the pain reliever ibuprofen over a long period of time can lead to a disorder in men called compensated hypogonadism. In their paper published ...

Nearly one-third of Canadians have used opioids: study

January 9, 2018
Nearly one in three Canadians (29 percent) have used "some form of opioids" in the past five years, according to data released Tuesday as widespread fentanyl overdoses continue to kill.

Growing opioid epidemic forcing more children into foster care

January 8, 2018
The opioid epidemic has become so severe it's considered a national public health emergency. Addiction to prescription painkillers, such as oxycodone and morphine, has contributed to a dramatic rise in overdose deaths and ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.