Category Archives: Muscular Dystrophy Treatment

DRYDEN, ONTARIO--(Marketwired - Aug. 1, 2014) - Muscular Dystrophy Canada and Safeway have launched their campaign to "Make Muscles Move" in support of those affected by neuromuscular disorders. Muscular Dystrophy Canada is partnering with Safeway stores in Northwestern Ontario and Western Canada from July 31 - August 15, 2014, to raise funds to support the Safeway Mobility Grant program, post-secondary scholarships for young adults and to fund important research. On Saturday August 10th, between 11:30am - 1:30pm, Rachel who is 20 years old and lives in Dryden and is affected by muscular dystrophy will be at the local Dryden store, "Helping to Make Muscles Move" with dedicated Safeway staff and their customers. Rachel says "Wow thank you Safeway Canada, and your mobility grant funding. Because of you I was assisted in purchasing a piece of equipment to assist in moving my legs. Since I received the equipment I have become much more independent. It has helped me maintain my leg strength and has allowed me to strengthen both my body and mind. This exercise device has also greatly reduced my leg pain caused by cramping. It allows me to exercise as I need in my home, so I now can … Continue reading →

A few days ago, I posted an article about a biotech company calledSarepta Therapeutics (SRPT) that was experiencing potentially crippling internal acrimony. Things seem to be settling down, allowing Sarepta to get back to work seeking regulatory approval for a highly anticipated medication. Heres the background, framed in human terms, from my earlier piece: Imagine for a harrowing moment that your child has a debilitating, deadly disease for which theres no cure. A biotech company has a drug that might arrest your childs decline, maybe keep him out of a wheelchair, possibly add years or decades to his life. It would be toughno, unbearableto watch infighting at the company impede access to the medication. Its heartbreaking, Mindy Leffler told me by phone. Her son Aidan, 11, has Duchenne muscular dystrophy (DMD), an inherited muscle-wasting disease that afflicts 1 in 3,500 boys born in the U.S. DMD robs its victims of the ability to walk in early adolescence. Pulmonary or other complications typically kill sufferers in their twenties.Leffler was reacting to a regulatory filing and related news reports about the firing of a key executive atSarepta [which is] is seeking Food and Drug Administration approval for a DMD treatment that could … Continue reading →

Parent Project Muscular Dystrophy logo. (PRNewsFoto/Parent Project Muscular Dystrophy) WASHINGTON, July 29, 2014 /PRNewswire-USNewswire/ --The House of Representatives today approved legislation to update the landmark Muscular Dystrophy Community Assistance, Research, and Education (MD-CARE) Act so the law continues getting results for all patients impacted by Duchenne and other forms of muscular dystrophy. The House approved the bill, H.R. 594, sponsored by Congressman Dr. Michael Burgess of Texas and Eliot Engel of New York, by unanimous voice vote. It must now clear the Senate to be signed into law by President Obama. Once that occurs, it will update the MD-CARE Act, which was enacted in 2001 and last updated in 2008. "Today's action is another significant milestone for all of us in the Duchenne and broader muscular dystrophy communities," said Pat Furlong, Founding President and CEO of Parent Project Muscular Dystrophy, the leading advocacy organization fighting to end Duchenne. "With this bill, we will ensure the MD-CARE Act keeps pace with the scientific and clinical needs of today and tomorrow, particularly for those with Duchenne and other forms of muscular dystrophy who are now living into adulthood." "We commend Congressmen Burgess and Engel for their tremendous leadership on behalf of all … Continue reading →

'Ministry can't interfere with judges' Orlando tells Senate (ANSA) - Rome, July 23 - Justice Minister Andrea Orlando told the Senate health committee Wednesday that the ministry can't interfere with a court ruling that forced a hospital to administer the controversial Stamina stem-cell treatment to an ailing boy. His statement came after a court ordered the treatment administered to a child suffering from muscular dystrophy at a hospital in northern Italy on Tuesday, despite the fact that it has been discredited by many in the scientific community. The Stamina treatment was administered on the orders of a Sicilian court after the hospital in Brescia decided to suspend the procedure because it had been called into doubt. The justice ministry "cannot interfere with the court's decisions. The judge has freedom of interpretation," Orlando told MPs. "However, no amount of court rulings can fill what is a legislative void on this issue," he added. The credibility of the Stamina treatment - which involves extracting bone-marrow stem cells from a patient, supposedly turning them into neurons by exposing them to retinoic acid for two hours, and injecting them back into the patient - has long been suspect, and last autumn the health ministry … Continue reading →

Dr.Dhanvanthri Premvel - Muscular Dystrophy - Treatment Part2 http://www.dhanvanthri.in, Dhavanthri Nilaym, Panchakarma, Muscular Dystrophy. By: Templecity Kuttisankar … Continue reading →

Dr.Dhavanthri Premvel - Muscular Dystrophy - Treatment part1 By: Templecity Kuttisankar … Continue reading →

(PRWEB) July 16, 2014 NIH awards $6.3 million grant to Seattle muscular dystrophy researchers A group of Seattle-based muscular dystrophy researchers has been awarded a prestigious $6.3 million grant from the National Institute of Health (NIH) to establish a Senator Paul D. Wellstone Cooperative Research Center. The group will focus on translating laboratory findings into clinical treatments for two of the most common inherited muscle diseases: Duchenne Muscular Dystrophy (DMD) and FSH Muscular Dystrophy (FSHD). This is welcome news to the local community of individuals with FSHD. Five years ago, if one asked a room full of scientists how the genetic mutation underlying FSHD caused the disease, the room would light up with fervor, imagination -- and contention. There were lots of ideas to explain why an anomalous deletion of junk DNA on chromosome 4 led to the progressive loss of skeletal muscle in patients with the disease, but no consensus on the cause. In 2004, the dearth of information about the disease prompted Terry and Rick Colella to form a non-profit organization, with a mission to stimulate research on FSHD, initially funding seed projects at the University of Washington, Seattle Childrens and the Fred Hutchinson Cancer Research Center. When … Continue reading →

A comedy night in support of FSHD Muscular Dystrophy will be held at Lafflines in New Westminsters Columbia Theatre on Saturday. #Standup4FSHD is being put on by Asifa Lalji, who suffers from the disease and is the sister of New Westminster Hyacks varsity football head coach and TSN reporter Farhan Lalji. The show will feature Vancouver comedian Carter Hortie and Californian Dino Archie, who has been doing standup in Vancouver for the last 11 years. FSHD causes muscles in the face, shoulder and back to waste away but can also affect other parts of the body. There is no treatment, but researchers are optimistic about some recent findings. Doors open at 6 p.m., with New Westminsters Mixed Company barbershop quartet greeting the patrons. Tickets are $20 (plus tax and service charge) and available at http://www.thecolumbia.ca or http://www.eventbrite.com. The event also has its own Facebook site at StandUp4FSHD and twitter address @#Standup4FSHD. Original post: Comedy night fundraiser for Muscular Dystrophy … Continue reading →

Sarepta Therapeutics, Inc. ( SRPT ) reported results through week 144 from a phase IIb study (Study 202: a long-term open-label extension study) on its lead candidate, eteplirsen, being developed for the treatment of Duchenne muscular dystrophy (DMD). Sarepta's shares tumbled 12.9% following the news. The study's 144 week data was comparatively weaker than the 120 week data reported in Jan 2014. Results from the study through 144 weeks revealed that patients under eteplirsen cohorts (30 mg/kg and 50 mg/kg) being able to perform a 6-minute walk test experienced a decline of 33.2 meters from baseline in walking ability. However, a statistically significant benefit of 75.1 meters was observed in modified Intent-to-Treat (mITT) population (n=6) compared with the placebo/delayed-treatment cohort (n=4), who started treatment with eteplirsen at week 25 after 24 weeks of placebo treatment. Meanwhile, Sarepta's previously reported 120-week data showed that patients under eteplirsen cohorts experienced a decline of 13.9 meters from baseline in walking ability as against a statistically significant benefit of 64.9 meters in mITT population compared with the placebo/delayed-treatment cohort, who started treatment with eteplirsen at week 25 after 24 weeks of placebo therapy. Sarepta shares had soared over 39% earlier this year after it … Continue reading →

For companies developing drugs for rare diseases, data in just a handful of patients can have enormous clinical implications and influence millions of dollars in market value. So it has been for Sarepta Therapeutics, developer of an experimental drug for Duchenne muscular dystrophy, a disease that affects about 1 in 3,500 boys worldwideor about 14,000 to 15,000 people in the U.S. Sarepta said Thursday a study of 12 boys suggests that its drug eteplirsen helped slow declines in walking ability for almost three years. Yet the stock sank, and was down about 24 percent mid-morning amid heavy selling in the broader market. One analyst said the data may call into question how well the drug works over longer periods. (For the latest stock quote for Sarepta, click here.) The results will be part of the company's application for approval of eteplirsen, which it plans to submit to the Food and Drug Administration this year, Sarepta Chief Executive Chris Garabedian said in an interview. The company also plans to start three broader studies, in over 100 patients, to provide further information. Read MoreSarepta gets path forward for muscular dystrophy drug "This dataset supports the treatment benefit we've seen over the last … Continue reading →