CARLSBAD, Calif., March 20, 2013 /PRNewswire/ --Isis Pharmaceuticals, Inc. (ISIS) announced that data from the Phase 1 study of ISIS-SMNRx in children with spinal muscular atrophy (SMA) were presented today at the 65th American Academy of Neurology (AAN) Annual Meeting by Dr. Claudia Chiriboga, from Columbia University Medical Center. In the presentation, Dr. Chiriboga reported that, in this single-dose, open-label study, ISIS-SMNRx was well tolerated in children with SMA at all dose levels tested and that improvements were observed in Hammersmith scores, a measure of muscle function, in a number of the children. SMA is a severe and rare genetic neuromuscular disease characterized by muscle atrophy and weakness and is the most common genetic cause of infant mortality. ISIS-SMNRx is an antisense drug designed to treat all types of SMA. "SMA is a devastating disease that results in severe muscle weakness and respiratory failure in infants and children. Current treatments are supportive and do not address the underlying genetic cause of SMA, the loss of SMN protein, which is critical to the health and survival of nerve cells responsible for neuromuscular growth and function. ISIS-SMNRx is designed to address the underlying genetic problem for patients with SMA by increasing the … Continue reading →

Get your name down for 'Town and Gown' 6:00pm Tuesday 19th March 2013 in News By Francesca Bardsley A MAN with a neuromuscular condition has urged runners to sign up for a race which raises cash for vital medical research. Management consultant Roger Wild, 62, has spinal muscular atrophy, which causes severe muscle weaknesses. He said the Town and Gown 10km run supported by the Oxford Mail on Sunday, May 12, provides much needed funds for the Muscular Dystrophy Campaign. He said: It is incredibly important to do the Town and Gown, particularly in a place like Oxford. Im sure academics and the citys population can really get to grips with the science of eradicating an incredibly debilitating condition, for which the Muscular Dystrophy Campaign is a driving force. The more we can do with cell research and treatments and the more people can run and raise money, the better. Mr Wild, who uses a powered wheelchair, has campaigned for better treatment for neuromuscular conditions. He is regularly invited to contribute to an all-party parliamentary group on the disease. Treatment varies depending on the priority given the conditions by 10 NHS strategic health authorities, which cover English regions, he said. … Continue reading →

Thirteen-year-old Austin and 10-year-old Max Laclaire play together with their Ben 10 action figures in Saxtons River. (Josh Stilts/Reformer file photo) BRATTLEBORO -- Laurie Webb wanted to put on a benefit for her grandsons with Duchenne Muscular Dystrophy. And, fortunately, she knows plenty of musicians. Webb was able to recruit three area bands -- Wyld Nightz, Pretty Daddy and Otis and the Elevators -- to perform at an event scheduled for Saturday at the American Legion. "Dancing for Duchenne" is set to begin at 7 p.m. and raise money for a trio of charities dedicated to helping those affected by DMD. Webb's daughter, Jenn McNary, has six children, two of whom suffer from Duchenne, an ultimately fatal degenerative muscle disease that affects roughly 20,000 children in the United States. Both Austin and Max Leclaire use the support of their family to live independent and productive lives, though their rare condition makes it a challenge. Max, 11, was eligible for a medical trial of Eteplirsen, a drug that greatly improved his quality of life. But Austin, 14, did not qualify and his condition has gotten progressively worse without the treatment. Max is able to walk while his older brother gets around … Continue reading →

CAMBRIDGE, Mass., March 20, 2013 /PRNewswire/ --DART Therapeutics Inc., an innovative, new-model biotechnology firm focused on developing therapies for Duchenne muscular dystrophy (DMD), announced today that it is developing a SARM drug candidate obtained from Belgium-based Galapagos NV. In early studies, the drug candidate, renamed DT-200, demonstrated significant potential to increase muscle size and strength. DT-200 could represent a new class of therapy for DMD, a muscle-wasting disease, and offer potential benefit for multiple neuromuscular diseases where improved muscle strength and function would be beneficial. Galapagos has provided the rights for its SARM drug candidate in DMD to the patient foundations Charley's Fund and theNash Avery Foundation, who co-founded DART Therapeutics. Terms of the rights transfer from Charley's Fund and Nash Avery to DART were not disclosed. Selective androgen receptor modulators--or SARMs--promote increased muscle mass and thereby strength--through normal androgenic pathways without the negative effects of oral androgenic steroids. DT-200 is an orally available SARM that has demonstrated potential to increase muscle size and strength in preclinical studies. In DMD, SARMs could be useful in halting progressive muscle loss by increasing the size and strength of diseased muscle. However, this remains an unexplored area of DMD therapy. Although the drug … Continue reading →

HACKENSACK, N.J., March 21, 2013 /PRNewswire-USNewswire/ --Parent Project Muscular Dystrophy (PPMD) has awarded University of Minnesota researcher Dr. Rita Perlingeiro a grant for $220,000 to develop a therapeutic approach for Duchenne muscular dystrophy (Duchenne) using a new type of "adult-derived" stem cell called "induced pluripotent stem cells" or "iPSCs." iPSCs can be created from non-controversial sources like skin, have the ability to multiply indefinitely, and can be coaxed to become many different kinds of tissue types. (Logo: http://photos.prnewswire.com/prnh/20100119/DC39975LOGO) Previously, Dr. Perlingeiro's group demonstrated that skin cells from donor mice that lack dystrophin could be converted into iPSCs, and then converted again into muscle stem cells with the mutation corrected using a modified viral carrier. When these corrected mouse stem cells were transplanted into recipient mice that lacked dystrophin, the mice showed improved muscle strength. Now Dr. Perlingeiro will repeat this process with funding from PPMD, this time using human skin cells from donors with Duchenne and using a safer method of correcting the dystrophin deficiency that does not rely upon random integration. The goal is to start laying the groundwork for an FDA-approved stem cell treatment for Duchenne. "Translating results in mice to treatments in humans is never straightforward," said … Continue reading →