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Archives
Category Archives: Muscular Dystrophy Treatment
Sarepta down as drug data disappoints
Posted: Published on July 10th, 2014
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
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Changing the care of muscular dystrophy
Posted: Published on July 4th, 2014
DAVID GARDNER-MEDWIN 1936-2014 David Gardner-Medwin was a naturalist, an expert on the 18th century Tyneside engraver Thomas Bewick, and a paediatric neurologist who specialised in muscular dystrophy. Muscular dystrophy is a genetic condition which usually affects young boys and for which until recently there has been no effective treatment. In the 1970s progressive muscle wasting often resulted in death before teenage years and in some parts of the country a fatalistic approach was taken to the disease with little attempt to treat the symptoms. Gardner-Medwin wnet to Newcastle, UK, in 1965 to work as a research fellow with Professor John, now Lord, Walton. There he set out to determine whether it was possible to detect which mothers carried the gene for muscular dystrophy by measuring electrical impulses from the muscles. As a result he spent long hours sitting with the mothers of disabled boys and was told of the many practical problems which affected their sons and how uncoordinated their care was. Such discussion spurred him to set up a multidisciplinary service with the child and the family at its centre. The resultant clinic became an exemplar of how to manage muscular dystrophy, and in 2009 the Muscular Dystrophy Campaign … Continue reading
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Schwartz: A Net Gain For A Tremendous Cause
Posted: Published on July 2nd, 2014
Nets' Radio Voice To Hold 4th Annual Dinner To Benefit His Foundation For FSHD July 2, 2014 7:00 AM Get our weekday morning briefs direct from the WFAN newsroom By Peter Schwartz More Columns There was a time when Chris Carrino was an avid sportsman. But during his college years at Fordham, his ability to compete athletically suffered a drop off as he began to feel the effects of weakening muscles, mostly in his shoulders and upper limbs. Then, in his early 20s, Carrino was diagnosed with facioscapulohumeral Muscular Dystrophy, a form of muscular dystrophy that affects thousands of adults and children throughout the world. Its a progressive disease for which there is no cure or treatment, but the radio voice of the Brooklyn Nets is dealing with it. I actually have been feeling pretty good, said Carrino. I try to live in the moment with it, but knowing that down the road its definitely a progressive thing that you have to deal with. You have to deal with the emotional effects of it and you have to deal with the physical effects of it. On July 17, Carrinos foundation for FSHD will hold its fourthannual dinner/dance at Russos on … Continue reading
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Youngsters at Somersham Primary School join disability games challenge from Sam Jerred, 9, who has Duchenne Muscular …
Posted: Published on July 2nd, 2014
Cambridge News Follow us on Thursday 3 Jul 2014 3:53 AM Written byJULIAN MAKEY SAM'S CHALLENGE: Sam Jerred, 9, centre, who has Duchenne Muscular Dystrophy, and schoolmates take part in a disability sports event at Somersham Primary School to raise money to tackle the condition 4 Images Pupils at Somersham Primary School have been learning about paralympic-style sports and games by taking part in Sams Sporting Challenge. They have also raised at least 500 for the charity Joining Jack to help combat Duchenne Muscular Dystrophy. Fellow pupil Sam Jerred, 9, who has the muscle-wasting condition, played a key part in leading the games. The children tried games like boccia, wheelchair football and running without using their arms, as well as experiencing using a wheelchair. Tracy Bauld, special educational needs co-ordinator, said: It has been fantastic, everyone has enjoyed themselves and Sam has gone home extremely exhausted. Read this article: Youngsters at Somersham Primary School join disability games challenge from Sam Jerred, 9, who has Duchenne Muscular ... … Continue reading
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Researchers Find Link To Failing Heart In Muscular Dystrophy
Posted: Published on June 26th, 2014
In a world first, researchers at The University of Western Australia have discovered a communication breakdown may be responsible for causing heart failure in people with muscular dystrophy. According to a study, mice that lacked the protein dystrophin had impaired functional communication between a calcium channel in the muscle cell membrane and the mitochondria that are responsible for producing the energy. Its understood that the hearts of muscular dystrophy patients fail but the mechanisms are unknown. It has also been previously reported that a lack of the protein dystrophin (responsible for muscular dystrophy) alters calcium channel function. In the latest study the researchers have identified a link between the channel and the mitochondria that involves dystrophin. In muscular dystrophy the functional communication between the channel and the mitochondria is impaired. We had previously shown that the L-type calcium channel could alter metabolic activity in the heart, Lead author Professor Livia Hool, explained. The L-type calcium channel is widely recognised as being important for regulating the heart beat and contraction. We had demonstrated an additional role for the channel in regulating metabolic activity. This involved altered calcium influx but part of the response also involved proteins that maintain cell structure. This … Continue reading
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Illinois Honors Parent Project Muscular Dystrophy's 20th Annual Connect Conference; Designates Week of June 23 as …
Posted: Published on June 24th, 2014
Parent Project Muscular Dystrophy logo. (PRNewsFoto/Parent Project Muscular Dystrophy) HACKENSACK, N.J., June 23, 2014 /PRNewswire-USNewswire/ --Parent Project Muscular Dystrophy (PPMD), the leading advocacy organization working to end Duchenne muscular dystrophy (Duchenne) will hold their 20th Annual Connect Conference in Chicago, Illinois, June 26-29, 2014. The State of Illinois will recognize the event by declaring the week of June 23 as Duchenne Muscular Dystrophy Awareness Week. With over 500 families and Duchenne experts expected to attend from around the world, this will be the largest Annual Connect Conference for PPMD to date. Illinois House Representative Darlene J. Senger of the 41st District will be presenting House Resolution #1112 which designates June 23-29, 2014 as Duchenne Muscular Dystrophy Awareness week in the State of Illinois. Ms. Senger, who is running for U.S. House of Representatives in November, will be presenting the Proclamation on Friday, June 27 at the Connect Conference. PPMD Founding President and CEO, Pat Furlong, was thrilled to hear about the Proclamation: "We carefully pick the city for our Annual Connect Conference and really wanted to find some place, not only convenient for our families, but special for our 20th meeting. Chicago is a favorite destination of our families and … Continue reading
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Isis testing safety of a new myotonic muscular dystrophy drug that targets toxic RNA
Posted: Published on June 22nd, 2014
US Isis Pharmaceuticals Inc (NASDAQ:ISIS) said yesterday it had started a Phase I trial of ISIS-DMPKRx, designed to lower the production of toxic dystrophia myotonica-protein kinase (DMPK) RNA in cells, including muscle cells, as a treatment for myotonic dystrophy type 1 (DM1). The company will get a milestone payment of USD 14 million (EUR 10.3m) from Biogen Idec for reaching this milestone. C. Frank Bennett, Isis senior vice president of research, said that ISIS-DMPK Rx is the first compound in the company's pipeline designed to target a toxic RNA, the first systemically administered drug to go into development from the partnerships with Biogen Idec and the second generation 2.5 drug to start clinical development. DM1, affecting some 150,000 people in the United States, Europe and Japan, is a rare genetic neuromuscular disease distinguished by progressive muscle atrophy, weakness and muscle spasms. DM1 is the most common form of muscular dystrophy in adults. DM1 patients have a genetic defect in their DMPK gene in which a sequence of three nucleotides repeats extensively, resulting in an abnormally long toxic RNA, which accumulates in the nucleus of cells and prevents the production of proteins necessary for normal cellular function. ISIS-DMPKRx is designed to … Continue reading
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You can still help Angelina and the MDA
Posted: Published on June 21st, 2014
Im going to tell you a story. Angelina was diagnosed with Spinal Muscular Atrophy (SMA) type 2 when she was 9 months old. The little girl, who lives with mom Erin and dad Frank in Liverpool, never sat up independently. She never crawled. She never walked. Shell have to rely on a power wheelchair to get around. When she was diagnosed, we were told it would be unlikely that we would see her second birthday, Erin said. But Angelina proved everyone wrong. She is now a spunky 2-and-a-half-year-old who is thriving, Erin said. With all the things we have been told she couldn't or wouldn't do, SMA has never stopped her from having the determination to do things she sees other kids her age doing. And as Angelina grows up, shes got a better and better chance of finding treatment as more and more research is completed in the field. Do you know what funds that research? Its you. Your donations to the Muscular Dystrophy Association are funding new research opportunities every day. And you helped me to provide some backing for those researchers with your donations for my bail for the MDAs 2014 Syracuse Lock-Up. According to the MDA, … Continue reading
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Building Act Exemption Unfair And Wrong
Posted: Published on June 19th, 2014
Building Act Exemption Unfair And Wrong The Muscular Dystrophy Association (MDA) this morning told the Local Government and Environment Select Committee that a proposed amendment to the Building Act, exempting owners from having to make their public buildings accessible, is unfair and wrong. MDA Chief Executive Chris Higgins told the Committee that the exemption would discriminate against people with disabilities by denying them access to public buildings, and would risk further marginalising them from their communities and society. Mr Higgins said that the exemption would be contrary to current legal requirements including the Building Act itself (which makes it mandatory for public buildings to be accessible to people with disabilities), the Human Rights Act (which makes it unlawful to deny a person access to a place because the person has a disability), and the United Nations Convention on the Rights of Persons with Disabilities (which states that the government must take measures to ensure people with disabilities have the right to access the physical environment on an equal basis with others). The proposed exemption will be unworkable Mr Higgins said. How will territorial local authorities possibly be able to decide whether being no longer earthquake-prone outweighs any detriment that is … Continue reading
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CureDuchenne Media Statement Regarding Prosensa Announcement of Regulatory Path Forward for Drisapersen as a Potential …
Posted: Published on June 3rd, 2014
NEWPORT BEACH, Calif.--(BUSINESS WIRE)--Insert a quote from Hans Schikan, Chief Executive Officer of Prosensa, after second paragraph of original release. The corrected release reads: CUREDUCHENNE MEDIA STATEMENT REGARDING PROSENSA ANNOUNCEMENT OF REGULATORY PATH FORWARD FOR DRISAPERSEN AS A POTENTIAL TREATMENT FOR DMD CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, issued the following statement today regarding the announcement by Prosensa Holding N.V. (NASDAQ: RNA) that the United States Food and Drug Administration (FDA) has outlined a regulatory path forward, under an accelerated approval pathway, for drisapersen, a potential treatment for Duchenne Muscular Dystrophy (DMD): This marks a significant milestone and gives hope to the boys and their families living with this devastating disease that a treatment is on the near-term horizon. Its been over 10 years since we began our partnership with Prosensa, and although the drug is not yet approved, these results validate our early efforts to fund exon skipping research, said Debra Miller, CEO and Founder, CureDuchenne. We applaud Prosensa for their tireless work in finding a treatment for the disease. CureDuchenne has been a loyal and excellent supporter since thevery early days of Prosensa. It is a great … Continue reading
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