Category Archives: Muscular Dystrophy Treatment

NEWPORT BEACH, Calif.--(BUSINESS WIRE)-- CureDuchenne Founder Debra Miller will speak at the Global Genes | R.A.R.E. Project Tribute to Champions of Hope benefit and RARE Patient Advocacy Summit on September 27-28, 2012, in Newport Beach, Calif. CureDuchenne is a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, a rare and devastating muscle disease that impacts one in 3,500 boys. Boys with Duchenne are usually diagnosed by age 5, are in a wheelchair by 12 and most dont live past their mid-20s. Miller will speak on a panel about nonprofit business models during the RARE Patient Advocacy Summit on September 28. Miller will also be a panelist following a Hollywood screening of Here. Us. Now., a rare disease documentary film commissioned by the Ewing Marion Kauffman Foundation. Directed by Emmy award-winning filmmaker Rudy Poe, Here. Us. Now. chronicles the story of how Hugh and Chris Hempel, parents with no medical backgrounds, use their entrepreneurial skills to find a breakthrough drug treatment for their twin daughters who are dying from a rare progressive neurological disease called Niemann Pick Type C. As the devastating effects of the disease begin to take hold, the Hempels enter a … Continue reading →

By Brenton Flynn | More Articles September 27, 2012 | Sarepta Therapeutics has a new drug in the works that could revolutionize treatment for Duchenne muscular dystrophy. This disease, characterized by a progressive weakening of the muscles, has a dismally low survival rate -- many who are afflicted live only into their teens. Results from a 36-week trial have produced promising data showing a meaningful decline in disease progression, and investors are anxiously awaiting 48-week data to be released in coming days. As a result, the stock has gone on a huge run, with shares more than tripling since early July. This could easily be a $500 million drug in the U.S., andGlaxoSmithKline is close on Sarepta's heels developing a treatment of its own. Sarepta is the one to watch in this space, particularly as the Oct. 9 meeting of the World Muscle Society draws closer. Check out the video below to learn more. When it comes to a do-or-die business, the biotech industry takes the cake. Arena Pharmaceuticals is another recent success story in the industry after gaining FDA approval for its innovative obesity drug. However, while the future looks bright, there are still plenty of obstacles ahead for … Continue reading →

Posted on September 26, 2012, Wednesday Che Azizan consoling his son Mohd Khairulnizam on board the Charlie C130 M30-06 after arriving at the Terminal II of Kota Kinabalu International Airport yesterday. KOTA KINABALU: The Royal Malaysian Air Force (RMAF) made a Mercy Flight for a patient, who is also a person with disabilities, from Labuan Hospital to the Queen Elizabeth Hospital (QEH) here yesterday. Mohd Khairulnizam Azizan, 18, was flown on board the Charlie C130 M30-06 at 2pm from Labaun airport and arrived at the Kota Kinabalu International Airport (Terminal II) at 2.40pm. According to Mohd Khairulnizams father, Che Azizan Mustapha, 42, his son suffers from muscular dystrophy since he was eight years old. I would like to take this opportunity to thank the Royal Malaysian Air Force (RMAF) for flying my son to Kota Kinabalu to get treatment at the QEH, he said when met yesterday. Meanwhile, Ministry of Defence (Sabah), media relation officer Lieutenant Mohd Hafiz Mohamed Sabilan said yesterdays Mercy Flight was the six made this year. He said the Mercy Flight was made after an approval was received from RMAF. > To enable your comment to be published, please refrain from vulgar language, insidious, seditious or … Continue reading →

NEW YORK (TheStreet) --I've written about Sarepta Therapeutics (SRPT) twice over the past month, with good reason. Despite a modest $327 million market capitalization, recent clinical data suggest that the company's lead drug candidate eteplirsen may be an effective treatment for Duchenne muscular dystrophy (DMD) -- a progressive disease that leaves patients wheelchair bound by their teens and dead shortly thereafter. My first article highlighted risks that I thought my colleague Adam Feuerstein missed in his bullish coverage. My second addressed comments I received from institutional investors who own Sarepta. They thought I was overly critical of eteplirsen even though I had declared Sarepta to be "a reasonable long position for biotech investors with a healthy appetite for risk." Last week, I learned an important tidbit that I don't think is widely known by the investment community: Sarepta's Phase IIb study of eteplirsen remained effectively blinded for far longer than 24 weeks. See if (SRPT) is in our portfolio That's important and potentially very good news for Sarepta. Before I explain why, let's recap the eteplirsen study to date: Continued here: A New Reason to be Bullish About Sarepta Therapeutics … Continue reading →

A fertility treatment which eliminates hereditary disease by engineering babies to carry healthy DNA from a third biological parent could be legalised next year. Members of the public are being asked whether families with a genetic risk of incurable conditions like muscular dystrophy should be allowed to use the DNA of a third party to create healthy children. Although the resulting babies would inherit a small fraction of their DNA from the donor and not their mother or father, the procedure would spare all future generations from a host of rare and debilitating conditions. The technique is currently forbidden as a treatment, but a public consultation launched today will help inform a decision by Jeremy Hunt, the health secretary, on whether the clinical benefits outweigh any ethical concerns. Experts accept the technique, which involves genetically modifying a human egg or embryo, enters "unchartered territory" and raises serious ethical questions. As well as the moral implications of engineering embryos, there are questions over how the procedure would impact on a child's sense of identity and whether they should be allowed to contact the donor later in life. Should Mr Hunt decide to give the treatment the green light the technique could … Continue reading →

ANN ARBOR, MI--(Marketwire - Sep 18, 2012) - Phrixus Pharmaceuticals, Inc., a specialty pharmaceutical company focused on innovative therapies for Duchenne muscular dystrophy (DMD) and heart failure, announced today that the United States Patent and Trademark Office (USPTO) issued a Notice of Allowance for U.S. Patent Application Serial Number 12/593,798, titled "Compositions and methods for the treatment of heart failure." The application includes claims intended to protect Phrixus's investigational drug Carmeseal (poloxamer-188) for the treatment of heart failure in a number of diseases with concomitant heart failure. A Notice of Allowance is issued after the USPTO makes a determination that a patent can be granted from an application. The issued patent would have a term that expires no earlier than in 2029. Such exclusivity would be separate from and in addition to exclusivity provided under the Orphan Drug Act and the Hatch/Waxman Act through the Food and Drug Administration for the treatment of DMD and other indications. Information on Phrixus's patents and pending applications in the United States is available on the Patent Application Information Retrieval system maintained by the USPTO at http://portal.uspto.gov/external/portal/pair. Phrixus has filed similar applications with the European Patent Office (EPO) and the Patent Cooperation Treaty (PCT). … Continue reading →

The most devastating news a parent can get is confirmation that their child has cancer, a disease that kills more children than cystic fibrosis, muscular dystrophy, AIDS, asthma and juvenile diabetes combined, according to the National Cancer Institute. Dalia Gonzalez knows what it feels like to receive this kind of diagnosis. Three years ago, her 7-year-old daughter, Violeta, was diagnosed with Leukemia. But soon after receiving the news from an oncologist at Loma Linda University Childrens Hospital, Gonzalez was greeted by representatives of the Childhood Cancer Foundation of Southern California, a Loma Linda-based non-profit that puts people like Gonzalez in touch with other families who have received similarly devastating news. The group not only helps the parents network, but it provides patients with helpful information, books and even financial assistance to help them cover their food and gasoline costs as they struggle to pay for the cost of cancer treatment. The Foundation also organizes social gatherings and special events to give families the emotional strength and support they need to get through the most physically and emotionally challenging period of their lives. The Foundations largest public event of the year takes place in Loma Linda Sunday, Sept. 9th, when the … Continue reading →

Public release date: 12-Sep-2012 [ | E-mail | Share ] Contact: Julie Langelier julie.langelier@ircm.qc.ca 514-987-5555 Institut de recherches cliniques de Montreal An important breakthrough could help in the fight against myotonic dystrophy. The discovery, recently published in the prestigious scientific journal Cell, results from an international collaboration between researchers at the IRCM, the Massachusetts Institute of Technology (MIT), the University of Southern California and Illumina. Their findings could lead to a better understanding of the causes of this disease. Myotonic dystrophy (DM), also known as Steinert's disease, is the most common form of muscular dystrophies seen in adults. This disorder is characterized by muscle weakness and myotonia (difficulty in relaxing muscles following contraction). It is a multi-system disease, typically involving a wide range of tissues and muscle. "We studied a specific family of proteins called muscleblind-like proteins (Mbnl), which were first discovered in the fruit fly Drosophila melanogaster," says Dr. ric Lcuyer, Director of the RNA Biology research unit at the IRCM. "These RNA-binding proteins are known to play important functions in muscle and eye development, as well as in the pathogenesis of DM in humans." Because of the extreme heterogeneity of clinical symptoms, DM has been described as one … Continue reading →

Gene Williams, co-founder and CEO of DART Therapeutics LLC Monday, September 10, 2012 DART Therapeutics isnt your ordinary therapeutics business. The Cambridge therapeutics startup focused on treatments for Duchenne muscular dystrophy(DMD), a fatal neuromuscular disorder with no current treatment affecting one in 3,500 boys worldwide, has made several new executive appointments to its leadership team. DART Therapeutics LLC has appointed its co-founder and executive chairman, Gene W. Williams as its CEO. In addition, the company has named Ernest Bush as chief scientific officer; Dr. Elliot Goldstein as its chief medical officer; and Cabot Brown as chief financial officer. Founded in 2010 by Williams and patient foundations in DMD, the Nash Avery Foundation and Charleys Fund, DART is a patient-funded biotech business focused on DMD and other rare diseases. Charleys Fund was founded by Tracy and Benjamin Seckler, parents of Charley Seckler who was diagnosed with DMD in 2004. The Nash Avery Foundation was created by Tom Wicka, whose son Nash was diagnosed with DMD. The patient-advocate groups created DART to make DMD research and drug development faster and less expensive, according to Seckler, Charleys dad and president of Charleys Fund. The families took matter into their own hands when they … Continue reading →

Gene Williams, co-founder and CEO of DART Therapeutics LLC Monday, September 10, 2012 DART Therapeutics isnt your ordinary therapeutics business. The Cambridge therapeutics startup focused on treatments for Duchenne muscular dystrophy(DMD), a fatal neuromuscular disorder with no current treatment affecting one in 3,500 boys worldwide, has made several new executive appointments to its leadership team. DART Therapeutics LLC has appointed its co-founder and executive chairman, Gene W. Williams as its CEO. In addition, the company has named Ernest Bush as chief scientific officer; Dr. Elliot Goldstein as its chief medical officer; and Cabot Brown as chief financial officer. Founded in 2010 by Williams and patient foundations in DMD, the Nash Avery Foundation and Charleys Fund, DART is a patient-funded biotech business focused on DMD and other rare diseases. Charleys Fund was founded by Tracy and Benjamin Seckler, parents of Charley Seckler who was diagnosed with DMD in 2004. The Nash Avery Foundation was created by Tom Wicka, whose son Nash was diagnosed with DMD. The patient-advocate groups created DART to make DMD research and drug development faster and less expensive, according to Seckler, Charleys dad and president of Charleys Fund. The families took matter into their own hands when they … Continue reading →