Category Archives: Muscular Dystrophy Treatment

Sarepta Therapeutics Inc.'s study of its experimental Duchenne muscular dystrophy drug eteplirsen may be too small for the faster approval the company seeks from regulators, according to an analyst who lowered her rating on the stock. Shares slide in premarket trading Tuesday. The Cambridge, Mass., company said Monday after markets closed that the U.S. Food and Drug Administration asked for more information from existing data on eteplirsen to help with its decision on whether to approve the treatment. Janney Capital Markets analyst Kimberly Lee said in a Tuesday morning research note she could not rule out the possibility that Sarepta's study of 12 patients treated with eteplirsen is too small for accelerated approval. Investors didn't seem optimistic, either. Sarepta shares slid 6.7 percent, or $2.64, to $36.60 before markets opened Tuesday. A Sarepta spokesman did not immediately return a call from The Associated Press seeking comment. The FDA grants accelerated approval to promising medical products designed to treat serious diseases. The program allows the agency to clear innovative drugs based on promising preliminary results. Duchenne muscular dystrophy is a fatal, genetic disease that causes increasing muscle weakness and affects one of every 3,500 boys worldwide. Sarepta said death usually occurs … Continue reading →

-1 of 2- 16 Apr 2013 05:15:00 UTC *DJ Independent DSMB Recommends Continuation of Santhera's Phase III Trial of Catena(R) in Duchenne Muscular Dystrophy (MORE TO FOLLOW) Dow Jones Newswires April 16, 2013 01:15 ET (05:15 GMT)- - 01 15 AM EDT 04-16-13 -2 of 2- 16 Apr 2013 05:15:00 UTC PRESS RELEASE: Independent DSMB Recommends Continuation of Santhera's Phase III Trial of Catena(R) in Duchenne Muscular Dystrophy Santhera Pharmaceuticals Holding AG / Independent DSMB Recommends Continuation of Santhera's Phase III Trial of Catena(R) in Duchenne Muscular Dystrophy . Processed and transmitted by Thomson Reuters ONE. The issuer is solely responsible for the content of this announcement. Liestal, Switzerland, April 16, 2013 - Santhera Pharmaceuticals (SIX: SANN) announced today that the Phase III DELOS study of orally administered Catena(R) in patients with Duchenne Muscular Dystrophy (DMD) successfully passed a planned futility and safety analysis. The independent Data Safety Monitoring Board (DSMB) for DELOS informed Santhera that the study has a reasonable chance of achieving its primary endpoint for improving or delaying the loss of respiratory function in Duchenne patients not using corticosteroids and since no safety issues were detected, recommended that the study should continue as planned. "I am very … Continue reading →

Liestal, Switzerland, April16, 2013 - Santhera Pharmaceuticals (SANN.SW) announced today that the PhaseIII DELOS study of orally administered Catenain patients with Duchenne Muscular Dystrophy (DMD) successfully passed a planned futility and safety analysis. The independent Data Safety Monitoring Board (DSMB) for DELOS informed Santhera that the study has a reasonable chance of achieving its primary endpoint for improving or delaying the loss of respiratory function in Duchenne patients not using corticosteroids and since no safety issues were detected, recommended that the study should continue as planned. "I am very enthusiastic about this news, which is important for the DMD community as it indicates that Catena eventually may evolve into a real treatment for all patients with this devastating disease. Importantly it suggests that the DELOS data are consistent with the outcome of the PhaseII DELPHI study, which showed that treatment with Catena resulted in a significant increase in peak expiratory flow and may improve early signs of respiratory weakness and dysfunction" commented Gunnar Buyse M.D., Ph.D., Professor of Child Neurology at the University Hospitals Leuven (Belgium) and Principal Investigator for the DELOS study. "Some promising therapies for DMD have failed to demonstrate clinical benefit in late-stage development, so it is … Continue reading →

GlaxoSmithKline Plc (GSK)s drisapersen achieved the primary objective of a mid-stage study, heating up competition with Sarepta Therapeutics Inc. (SRPT) in the race for a drug that could reverse the debilitating effects of Duchenne muscular dystrophy. Patients taking drisapersen showed a clinically meaningful difference from those on placebo after 48 weeks of treatment, according to an abstract of the study results posted on the website of CureDuchenne, an investor in Glaxos partner on the drug, Dutch biotechnology company Prosensa Therapeutics BV. Glaxo will present the data at a genetics conference in Cold Spring Harbor, New York, tomorrow. Duchenne muscular dystrophy is a neuromuscular disease with no known cure that affects one in 3,500 newborn boys. Drisapersen has been designated by U.S. and European regulators as an orphan drug, and may eventually reach 500 million pounds ($766 million) in sales if proven to be effective and approved for commercialization, according to Fabian Wenner, an analyst at Kepler Capital Markets in Zurich. We are very pleased to report that the results are clinically significant, CureDuchenne, a non-profit organization based in Newport Beach, California, said on its website. Its been a long 10 years and the drug is not yet approved, but we … Continue reading →

BOSTON (TheStreet) -- The race between Sarepta Therapeutics (SRPT) and GlaxoSmithKline (GSK) to develop the first new therapy for Duchenne muscular dystrophy just took an interesting twist. Glaxo decided -- somewhat abruptly -- to present results from its randomized phase IIb study of drisapersen at a research conference on Thursday. This is the first time Glaxo is sharing data from any placebo-controlled study of drisapersen in Duchenne muscular dystrophy (DMD) patients. The timing is odd for a couple of reasons: Glaxo and partner Prosensa have said several times in the past that no data from this drisapersen study would be announced until the third quarter (when the ongoing phase III study was expected to be completed.) Glaxo also chose a somewhat obscure venue to present the phase IIb drisapersen data Thursday -- the RNA & Oligonucleotide Therapeutics conference at Cold Spring Harbor Laboratories. A more high-profile venue would have been the The Muscular Dystrophy Association's Scientific Conference, scheduled for April 21-24. Here is where you can let your speculative juices flow. Is Glaxo trying to hide negative drisapersen data? Or, are the drisapersen data so positive that Glaxo is rushing to get the news out ahead of the expected announcement … Continue reading →

CAMBRIDGE, MA--(Marketwired - Apr 5, 2013) - Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced updated data from Study 202, a Phase IIb open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD). Results at 74 weeks showed a continued stabilization of walking ability in eteplirsen-treated patients evaluable on the 6-minute walk test (6MWT). As previously reported, Study 202 met its primary endpoint of increased novel dystrophin as assessed by muscle biopsy at week 48 and is now in the long-term extension phase in which patients continue to be followed for safety and clinical outcomes. Eteplirsen is Sarepta's lead exon-skipping compound in development for the treatment of patients with DMD who have a genotype amenable to skipping of exon 51. After 74 weeks, patients in the 30 mg/kg and 50 mg/kg dose cohorts who were able to perform the 6MWT (modified Intent-to-Treat or mITT population; n=6) showed a statistically significant treatment benefit of 65.2 meters (p 0.004) when compared to the placebo/delayed-treatment cohort (n=4). The eteplirsen-treated patients in the mITT population demonstrated less than a 5 percent decline (13.4 meters) from baseline in walking ability. After experiencing a substantial decline earlier in the … Continue reading →

Today's most viewed articles EXETER Today, April 5 from 6 to 8 p.m., at The Provident Bank in Exeter (95 Portsmouth Ave.), eighth-grader Rebecca Gowing and art teacher Crystal Buswell will host an art show, reception and silent auction to raise awareness of Duchenne Muscular Dystrophy. Duchenne Muscular Dystrophy is a little known and often misunderstood genetic disorder, and tonight's event looks to raise funds on behalf of Parent Project Muscular Dystrophy (PPMD). Art for a Cure art show and silent auction When: From 6-8 p.m., Friday, April 5 Where: The Provident Bank, 95 Portsmouth Ave., Exeter More: facebook.com/ArtToCureDMD There are at least two young men third-grader Cameron Yurik of Brentwood and fifth-grader Lucas Currier of Newfields in our community with this progressive muscle disorder. PPMD is a national nonprofit organization started in 1994 by parents of young men diagnosed with Duchenne. Gowing is actively involved with PPMD and hopes to inspire the Seacoast community to come out, have fun, and purchase beautiful artwork in support of PPMD. The art show will feature nature-themed two-dimensional artwork by both students and professional artists. Students in the third and fifth grade classes of the young men who have Duchenne will be creating … Continue reading →

Eteplirsen keeps up its steady walk toward approval. Sarepta Therapeutics (NASDAQ: SRPT) reported the latest clinical study results today for its Duchenne muscular dystrophy, or DMD, drug. Shares in the biotech are up nearly 5% as the trading day winds down. Latest resultsSarepta's news related to results from 74 weeks into a phase 2b study of eteplirsen. Patients taking the drug demonstrated continued stabilization of walking ability on the six-minute walk test, a standard assessment of walking capability and clinical function for neuromuscular diseases. As with earlier points in this study, DMD patients taking eteplirsen showed significant improvement in walking ability compared to patients on placebo. This was true for groups taking the 30 mg/kg and 50 mg/kg dosages of the drug. Eteplirsen also continued to be well-tolerated. Sarepta reported that no patients experienced significant treatment-related adverse events or any serious adverse events. At week 62, one patient had a temporary elevation of urine protein, but that was resolved and the patient stayed in the study with no further problems. Chris Garabedian, Sarepta's president and CEO, said that the company was "encouraged to see a continued stabilization of walking ability in patients treated with eteplirsen for nearly one and a … Continue reading →

Wonder drug hope thanks to Town and Gown run 5:00pm Wednesday 3rd April 2013 in News By Katriona Ormiston, Reporter. Call me on (01865) 425426 A SCIENTIST working to create a pill to treat muscular dystrophy is backing a fundraising run for research. Professor Dame Kay Davies is urging people to sign up to the Oxford Town and Gown run in Oxford city centre on May 12. The 10k run raises money for the Muscular Dystrophy Campaign, which is funding her work. The degenerative muscle-wasting disease spreads to the legs and arms, and sufferers usually die in their 20s after their respiratory muscles fail. Prof Davies, from Oxford Universitys Department of Physiology Anatomy and Genetics, said: I have been working on this for 30 years and I am not going to retire until I find an effective treatment for this terrible disease. We are so close now. Ten years ago I didnt think there was a chance we would be at the stage we are now, its very exciting. We have found a drug which in mice shows substantial improvements. The question now is will it work with patients? What we are hoping it will do is significantly slow disease … Continue reading →

muscular dystrophy treatment in varma successful-treatment.blogspot.in. By: varma4md … Continue reading →