Category Archives: Muscular Dystrophy Treatment

Sarepta Therapeutics Inc. (SRPT), a developer of an experimental drug for Duchenne muscular dystrophy, may seek advisers to find an overseas partner for the therapy, Chief Executive Officer Christopher Garabedian said. The biotechnology company is talking with more than a dozen drugmakers about a partnership for international sales of eteplirsen, Sareptas medicine for the rare muscle-wasting disease, Garabedian said in an interview at the JPMorgan Chase & Co. health-care conference in San Francisco. Cambridge, Massachusetts-based Sarepta is considering hiring an advisory firm, such as Centerview Partners LLC or JSP Partners, to help with the talks. The company isnt for sale, he said. While successful testing of eteplirsen may make Sarepta, a 32-year-old company with no marketed products, an acquisition target, Garabedian said he wants it to become a rival to large biotechnology companies such as Gilead Sciences Inc. (GILD), the worlds biggest maker of AIDS medicines and Celgene Corp. (CELG), the maker of the $3 billion cancer drug, Revlimid. Im building a team that knows how to create a successful global biopharmaceutical company, Garabedian said. Im hiring people from Genzyme, Gilead, Celgene, Shire, and Vertex. Those people like myself want to work on the next break-out biotech. Duchenne muscular dystrophy … Continue reading →

SAN DIEGO--(BUSINESS WIRE)-- Ligand Pharmaceuticals Incorporated (LGND) announced today that it has received a milestone payment of 620,000 shares of common stock in newly public partner Retrophin, Inc. (RTRX). The milestone arose under the previously executed license agreement for the development and commercialization of Retrophins lead clinical candidate RE-021, formerly known as DARA (a Dual Acting Receptor Antagonist of Angiotensin and Endothelin receptors) and was triggered by the completion of Retrophins merger with Desert Gateway, Inc. and its transition to a publicly traded company. Ligand will record milestone revenue equal to the estimated fair value of the shares received, which will be determined by an independent valuation firm. The shares issued to Ligand represent approximately 7% of Retrophins outstanding capital stock and may be subject to certain trading restrictions. RE-021 is in development for the treatment of focal segmental glomerulosclerosis (FSGS), a rare disease that attacks the kidneys filtering system (glomeruli), causing serious scarring, progressive kidney function degeneration and rapid loss of the kidneys. FSGS is one cause of a serious condition known as Nephrotic Syndrome. An estimated 50,000 patients in the United States suffer from FSGS, with most patients diagnosed as children or young adults. Ligand believes that Retrophin … Continue reading →

Researchers have discovered what could be the first step in preventing the onset of a heart complication that kills one out of every five people suffering from Duchenne muscular dystrophy (DMD). The results were just published in STEM CELLS Translational Medicine. Durham, NC (PRWEB) January 08, 2013 In DMD, the most common form of muscular dystrophy, patients lack a large, rod-like protein called dystrophin located primarily in muscles used for movement and in heart muscle. The dystrophin is part of a group of proteins that acts as an anchor, connecting each muscle cell's structural framework with the lattice of proteins and other molecules outside the cell. Without dystrophin, many of the muscle cells in the heart are damaged, subsequently die and are replaced by connective tissue. Many Duchenne MD patients suffer from dilated cardiomyopathy (DCM), a condition in which the chambers of the heart are enlarged and weakened, explained the studys lead author, Suzanne Berry, Ph.D. As a result the heart cant efficiently pump blood to the body and many patients eventually die. We hypothesized that mesoangioblast stem cells (ADM) found in the walls of large blood vessels, in this case the aorta, would restore dystrophin and therefore alleviate or … Continue reading →

WALTHAM, Mass.--(BUSINESS WIRE)-- Repligen Corporation (RGEN) announced today that it has entered into an exclusive worldwide licensing agreement with Pfizer Inc. to advance Repligens spinal muscular atrophy (SMA) program, originally in-licensed from Families of SMA (FSMA). The SMA program includes RG3039, a small molecule drug candidate in clinical development for SMA, as well as backup compounds and enabling technologies. Under the terms of the agreement, Repligen is entitled to receive up to $70 million from Pfizer, commencing with an upfront payment of $5 million and total potential future milestone payments of up to $65 million as well as royalties on any future sales of SMA compounds developed under the agreement. SMA is an orphan neurodegenerative genetic disease that presents early in life. This agreement is consistent with the strategic decision we announced in August 2012 to focus Repligens internal efforts on the growth of our bioprocessing business, while seeking external partners for our therapeutic development programs, said Walter C. Herlihy, Ph.D., President and Chief Executive Officer of Repligen. We believe this collaboration with Pfizer, a leading pharmaceutical company with specialized efforts in orphan and genetic diseases, has the potential to accelerate the development of therapies for SMA. There is a … Continue reading →

The Great Idea of Humanitarianism and Profitable Muscular Dystrophy Clinic from Private Practises. Hello everybody around the world.I hope all of you have a wonderful Christmas and best wishes in the coming new year. My name is Dr.Imada Laura HP I am World Expert Muscular Dystrophy with International scope,most of my work online (consultation online via email dr.laurainternational@yahoo.com),possibility offline too.It mean I am willing to help all the patients around the world. I am Medical Doctor with 15 years work in every health fields.I was also accepted as Doctor on call mental illness at California on February 2011,but I had to come back to Indonesia. Right now I open my private practises at Jl.Kebagusan Raya Gg Kriep no 53.Jagakarsa.Jakarta Selatan.Indonesia.Telephone 62217271766 or my cellphone 6285219268188.My email dr.laurainternational@yahoo.com The programs of my private practises are : 1.General Treatment for all kind of disease 2.Muscular Dystrophy Treatment 3.Physiotherapy 4.Body mind healing 5.Aquatic therapy 6.Career Development for disability that influence the process of healing with hope the patients can get cure as soon as possible. 7.Minor Surgery 8.Machine Migun (combination chiropatrick,accupressure,accupuncture by machine use for pain treatment,reduce stress,treatment for the back pain,functional back,anatomic to make normal the back,stroke,etc) 9.Imunisation,Contraception 10.Healing for long distance … Continue reading →

CLEVELAND, Dec. 12, 2012 /PRNewswire/ -- Milo Biotechnology today announced its AAV1-FS344 has been granted Orphan Drug designation from the FDA's Office of Orphan Products Development for treatment of Becker and Duchenne muscular dystrophy. AAV1-FS344 is a gene therapy-delivered myostatin inhibitor that increases muscle strength. The program is currently in a Phase I/II trial at Nationwide Children's Hospital in adult patients with Becker muscular dystrophy and inclusion body myositis, a trial funded by the foundation Parent Project Muscular Dystrophy. "The Orphan Drug designation will provide incentives for Milo Biotechnology's continued development of the therapy in rare muscular dystrophies with the greatest medical need," said CEO Al Hawkins. "We hope that our potent muscle strengthening approach will someday augment emerging genetic strategies to transform care for these intractable diseases." Duchenne muscular dystrophy is the most common inherited myopathy, caused by the absence of dystrophin protein. Becker muscular dystrophy, a less severe form of Duchenne muscular dystrophy, is caused by reduced levels of dystrophin protein. With both types, patients experience progressive muscle weakness, and cardiac and respiratory complications. The Orphan Drug Act (ODA) grants special status for products to treat rare diseases. Orphan designation qualifies the sponsor of the product for a … Continue reading →

SEATTLE, Dec. 10, 2012 /PRNewswire/ --Fred Hutchinson Cancer Research Center and GlaxoSmithKline PLC (GSK) today announced a partnership to develop therapeutics to treat an inherited form of muscular dystrophy. The goal of the new agreement is to develop a small-molecule-based medicine to potentially reverse facioscapulohumeral muscular dystrophy, or FSHD, by inhibiting the activity of a protein that is incorrectly expressed by the DUX4 gene in people with the disease. The protein activity is what damages muscle cells and leads to progressive muscle weakness and atrophy in FSHD patients. The genetic and disease mechanisms of FSHD were discovered by an international team of scientists led by Stephen Tapscott, M.D., Ph.D., a member of the Fred Hutch Human Biology Division, in a series of studies published between 2010 and early 2012. Tapscott will lead the Fred Hutch work in the GSK collaboration. The team's discoveries also have implications for developing cancer immunotherapies because researchers also discovered that DUX4 regulates cancer/testis antigens. Cancer/testis antigens are encoded by genes that are normally expressed only in the human germ line but are also abnormally expressed in various tumor types, including melanoma and carcinomas of the bladder, lung and liver. This knowledge will give researchers a … Continue reading →

Shares of Sarepta Therapeutics Inc. slid Friday, even though the company announced more positive results from a study of its Duchenne muscular dystrophy drug eteplirsen. THE SPARK: The Cambridge, Mass., company said Friday patients treated with eteplirsen for more than a year did better on a six-minute walking test than those treated with a placebo for 24 weeks and the drug for 38 weeks in the extension of a mid-stage trial. THE BIG PICTURE: Duchenne muscular dystrophy is a genetic disease that causes increasing muscle weakness. It is caused by a lack of the protein dystrophin, and it affects only boys. The condition occurs in about 1 of every 3,600 male infants. The National Institutes of Health say patients typically die before the age of 25. Sarepta's stock nearly tripled in value in early October after it said eteplirsen slowed the progress of the disease and increased patients' dystrophin levels. The shares also jumped in July when the company announced some initial results of the trial extension. Eteplirsen is the most advanced drug candidate for Sarepta, formerly known as AVI BioPharma. THE ANALYSIS: Steve Brozak of WBB Securities said the stock is suffering a little bit from "news fatigue." "This … Continue reading →

SOUTH PLAINFIELD, N.J., Dec. 6, 2012 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced that the European Medicines Agency (EMA) has validated a Marketing Authorization Application (MAA) seeking conditional approval for ataluren, an investigational new drug for the treatment of patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). Validation of the MAA confirms that the submission is complete and begins the EMA's Committee for Human Medicinal Products' (CHMP) review process. Ataluren is the only treatment currently in clinical development targeting the cause of disease in patients with a nonsense mutation. (Logo: http://photos.prnewswire.com/prnh/20010919/PTCLOGO) "Ataluren is a promising potential therapy for nonsense mutation Duchenne muscular dystrophy," stated Dr. Thomas Voit, Medical and Scientific Director, Institut de Myologie. "PTC has developed a standard for DMD clinical trials and now the DMD community can share in the achievement of the first MAA ever filed for DMD. We appreciate PTC's commitment to the clinical development of ataluren for this severe disorder for which only palliative treatment options currently exist." The Marketing Authorization Application's submission was accepted by EMA for review on the basis of a 48-week, 174-patient Phase 2b study showing that nmDMD patients treated with ataluren (10, 10, 20 mg/kg given daily) walked on … Continue reading →

Furlong Will Address Role of Advocacy in Facilitating Basic Scientific Research HACKENSACK, N.J., Dec. 3, 2012 /PRNewswire-USNewswire/ --Parent Project Muscular Dystrophy (PPMD) founding president and CEO Pat Furlong, has been invited to speak today at the Institute of Medicine's (IOM) Roundtable on Translating Genomic-Based Research for Health. IOM is holding a public workshop today in Irvine, CA titled "Improving the Efficiency and Effectiveness of Genomic Science Translation." (Logo: http://photos.prnewswire.com/prnh/20100119/DC39975LOGO) Pat is a member of IOM's Committee on Pediatric Studies and is thrilled to be participating in the discussion "The Role of Advocacy in Facilitating Basic Scientific Research" at this important roundtable. "Advocacy has never played a more important role in the fight to cure rare disease, than it does today. As a patient advocacy group, it is critical for our voices to be heard in Washington. This is what IOM stands for and why they were created. To help amplify voices like PPMD's. They also understand that it takes creativity, thinking outside of the box, to reach the ears of the federal government and I am humbled that they have chosen me to participate in this discussion. PPMD has done and continues to do everything it can to end Duchenne … Continue reading →