Category Archives: Muscular Dystrophy Treatment

The FDAs decision to give an experimental drug for Duchenne muscular dystrophy another chance is rekindling hope in parents in the Bay State and beyond that they might be able to save their children from the worst effects of the fatal disease. The Food and Drug Administration has cleared the way for Cambridge-based Sarepta Therapeutics to apply for approval of eteplirsen, a drug that treats DMD. Its a move Sarepta has been awaiting and DMD families have been demanding. This is certainly a step in the right direction, said Tracy Seckler, whose son Charley suffers from the muscle-wasting disease. Its hard to express extreme joy at the FDA doing its job properly and doing what should have been done already, but under the circumstances its encouraging. The FDA previously had stalled approval of eteplirsen over concerns about its safety and efficacy. The drug has been tested in only 12 boys, but it appears to be effective for them. Boys with DMD lose mobility over time and typically dont live past their 20s. There are about 18,000 to 20,000 DMD patients in the United States. The FDA is fully committed to make safe and effective drugs available for patients with Duchenne … Continue reading →

Sarepta Therapeutics Inc's shares soared 64 percent after it said that the U.S. Food and Drug Administration indicated an alternate path to approval for the company's experimental muscle disorder drug. The news provides a fresh lease of life for Sarepta, whose plans for a marketing approval the regulator deemed "premature" late last year, citing insufficient trial data. The agency has now indicated that safety and efficacy data from studies without placebo groups could support an approval application, analysts said. The regulator had earlier said that the company would need to compare the drug, eteplirsen, with a placebo. Patients treated with the drug in the new trials can now be compared with historical data from other patients with Duchenne muscular dystrophy (DMD), analysts said. Sarepta plans to file for approval by the end of the year and expects to get marketing nod in the second half of 2015. "Currently we have 2018 as the year of launch for eteplirsen, but this timeline could shave as much as 2+ years off that projection," Cowen & Co analyst Edward Nash said in a note. Sarepta is developing eteplirsen as a treatment for DMD, a degenerative disorder that hampers muscle movement and affects one … Continue reading →

By Estel Grace Masangkay Sarepta Therapeutics, a company focused on developing RNA-based treatments, announced its intent to file a New Drug Application for eteplirsen for the treatment of Duchenne muscular dystrophy (DMD) by the end of 2014. Eteplisen is the companys lead exon-skipping drug candidate under development for the treatment of patients with DMD who have a genotype disposed to skipping of exon 51. The company said it reached the decision to file an NDA by the end of the year based on a guidance letter from the FDA. According to the agency, an NDA for eteplirsen could be filed under a potential Accelerated Approval pathway. The FDA also provided guidance on an open-label, historically controlled confirmatory study of the drug as well as initial guidance on a placebo-controlled study on one or more of the companys follow-on DMD drug candidates. Chris Garabedian, president and CEO of Sarepta Therapeutics, said, As we announce our plan to submit an eteplirsen NDA by the end of 2014, we are very pleased with the detailed guidance that the FDA has provided us on a potential eteplirsen approval pathway and their support of a historically controlled eteplirsen confirmatory study. We also appreciate that the … Continue reading →

Published: Monday, April 21, 2014, 10:03p.m. A new drug designed to slow the progression of Duchenne muscular dystrophy, a devastating disease that overwhelmingly affects boys and typically leaves them needing wheelchairs by their teens and dead in their 20s, has received a long-awaited boost from the Food and Drug Administration. Massachusetts-based Sarepta Therapeutics said Monday that FDA officials detailed a potential path forward for the drug, eteplirsen, and indicated a willingness to consider it for accelerated approval. The news puts eteplirsen one step closer to becoming the first approved treatment for Duchenne, a disease that affects about one in 3,500 boys globally. 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 Chris Garabedian said. I think that's a huge win for the [Duchenne] community and for Sarepta. The move follows a tumultuous year in which the FDA initially appeared headed toward swift approval of eteplirsen based on promising results from a trial involving 12 young boys, whose muscular deterioration seemed virtually halted by the drug. But the agency hit the pause button in November, saying it would be premature for Sarepta … Continue reading →

Sarepta Therapeutics (SRPT) Inc., a drug developer with no products on the market, rose to its highest value in five months after saying it plans to submit its Duchenne muscular dystrophy treatment for approval this year. Sarepta increased 39 percent to $33.98 at 4 p.m. New York time, its highest closing price since Nov. 11. On Nov. 12, Sarepta shares fell the most in 16 years after the Food and Drug Administration had called an application to sell the experimental eteplirsen compound premature. The FDA has provided new guidance that will enable the submission of the drug by the end of 2014, the Cambridge, Massachusetts-based company said today in a statement. We are excited to have guidance from the FDA that allows us to move quickly,said Edward Kaye, chief medical officer at Sarepta. Sarepta anticipates the FDA may call advisers to discuss eteplirsen in the first half of 2015 and approve the drug by the second half of next year, Robyn Karnauskas, an analyst at Deutsche Bank AG, said in a note to clients today. Duchenne muscular dystrophy is a genetic disorder that mainly affects boys between ages three and five that causes progressive muscle degeneration and weakness, according to … Continue reading →

The FDA made clear that its guidance doesn't reflect how it will vote on Sarepta's drug, and said the company should be prepared for an advisory committee meeting to evaluate the application before the agency makes its decision, Sarepta said. Read MorePfizer weighs massive bid for rival AstraZeneca "They haven't reversed their decision from November," Sarepta Chief Executive Chris Garabedian said in a telephone interview. "They've reinforced the questions and concerns they had of being able to accept an NDA and consider accelerated approval of just the data set they had in-house, but evolved to say, 'If you give us a little more data, it should be fileable.'" Garabedian is expected to appear on "Street Signs" at 2 p.m. The last study Sarepta conducted involved just 12 patients, a very small set for the FDA to consider. But Duchenne muscular dystrophy is a rare, fatal condition for which no other treatment options exist. And the company argues its data, most recently reported out to 120 weeks in these 12 patients, show the drug helps stem declines in walking ability. See more here: FDA to review Sarepta drug … Continue reading →

By Nathania Johnson 04/18/2014 at 10:15 AM EDT Max Vertin, wearing his Iron Man costume, prepare to enter a wind tunnel Brennan Linsley/AP Thanks to the Make-A-Wish foundation, the 8-year-old Nebraska boy visited an indoor wind tunnel, which simulates skydiving conditions, and flew like his favorite superhero, Iron Man. Emulating his hero was just the beginning. Max received a surprise visit from the armored billionaire genius, who was decked out in his full red and gold costume. He presented Max with his very own Iron Man suit. "This is awesome. This is the break we needed. It's nice not be at the doctor's and getting them shots. We can just have fun," sister Lexi told KUSA. Max transformed into such a convincing superhero that by the time his limo pulled away from the wind tunnel facility, a crowd was cheering "Iron Max." It looks like Tony Stark has some competition. Brown has battled brain cancer for two years, but the tumor's persistent growth has forced her to leave school. Thats when Brown's friends decided to throw her a "high school in a day" experience. The teen participated in a private graduation ceremony and was crowned prom queen. When her friends … Continue reading →

Contact Information Available for logged-in reporters only Newswise Sometimes known as Kennedys disease, spinal and bulbar muscular atrophy (SBMA) is a rare inherited neuromuscular disorder characterized by slowly progressive muscle weakness and atrophy. Researchers have long considered it to be essentially an affliction of primary motor neurons the cells in the spinal cord and brainstem that control muscle movement. But in a new study published in the April 16, 2014 online issue of Neuron, a team of scientists at the University of California, San Diego School of Medicine say novel mouse studies indicate that mutant protein levels in muscle cells, not motor neurons, are fundamentally involved in SBMA, suggesting an alternative and promising new avenue of treatment for a condition that is currently incurable. SBMA is an X-linked recessive disease that affects only males, though females carrying the defective gene have a 50:50 chance of passing it along to a son. It belongs to a group of diseases, such as Huntingtons disease, in which a C-A-G DNA sequence is repeated too many times, resulting in a protein with too many glutamines (an amino acid), causing the diseased protein to misfold and produce harmful consequences for affected cells. Thus far, human … Continue reading →

CHICAGO, (PRWEB) April 15, 2014 The Muscular Dystrophy Association, the largest nonprofit funder of neuromuscular disease research in the United States, has awarded funding for 38 new research grants totaling $3.6 million. The awards will be made to researchers seeking promising treatments and cures for muscle diseases within MDA's program. The new grants are in addition to more than 200 research projects underway this year across 12 countries. Funding of the new grants will begin May 1 and continue for two to three years, depending on the individual grant request. "Our mission is to save and improve lives, and these new grants provide more insight into the causes of these diseases, and represent the best in current possibilities to help lead us to treatments and cures," said Valerie Cwik, M.D., MDA's Executive Vice President and Chief Medical & Scientific Officer. "These grants build on the remarkable progress we're making with more new drug discovery and clinical trials underway than ever before." For more than 60 years, MDA has funded research to investigate the causes of neuromuscular disease as well as research seeking treatments and cures. The organizations specialized clinic program is at the forefront of research and treatment methods and … Continue reading →

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