Category Archives: Muscular Dystrophy Treatment

Marty Karlin was taking boxes of his newly marketed "ShtankOut" toys to a UPS store when an employee asked him what he was sending out. The small, colorful, smiley characters that eliminate odors caught her eye. They would be perfect for parents looking to throw a little something extra in a package for a college-age son or daughter, she told him. Why not sell them in the store? Karlin, a Naperville resident, had just found another market in a growing number of retail outlets selling his new fundraising tool for muscular dystrophy. A portion of the $4.99 to $5.99 price for every Miracle Monkey, Funny Face and their pals goes toward research for Duchenne muscular dystrophy, the muscle-wasting disease that Karlin's 10-year-old son, Ryan, was diagnosed with five years ago. Karlin and his wife, Geri, hope their efforts will help keep Ryan doing the activities he loves beyond current expectations. Boys with Duchenne muscular dystrophy normally become wheelchair dependent before age 13 and many do not live beyond their mid-20s. As the disease progresses, it leaves them unable to move their legs, arms and hands, and eventually attacks the heart and lungs. "Things need to happen quickly," Geri says. "You … Continue reading →

By MONICA DRAKE - monica.drake@oakpress.com; Twitter: @monica_adele Walled Lake Student Council is hosting a dinner benefit for Commerce Township resident Joseph Penrod, 10, (left) who suffers from Duchenne Muscular Dystrophy, in order to help make his home accessible. He is pictured with his mother Marissa. By the age of 5, most children spend time running, jumping and skipping so Commerce Township resident Marissa Penrod knew something was wrong when her son couldnt do these things. In 2008, Joseph Penrod, now 10, was diagnosed with Duchenne Muscular Dystrophy, which is the most common progressive degenerative muscle disease in boys. Although he can stand on his own, Joseph, a fourth-grader at Oakley Park Elementary, spends most of his time traveling by scooter. My first reaction when he was diagnosed was fear. ... But I made a commitment within days of his diagnosis that we would have to do everything we could to change the future of it, said his mother. Penrod created the nonprofit Team Joseph shortly after her sons diagnosis in hopes to find a cure for the disease. Last year, almost $300,000 was donated through the organization toward medical research at Phrixus Pharmaceuticals in Ann Arbor and at the universities … Continue reading →

Reading firefighters will be out at prominent city intersections Friday asking motorists to help "Fill the Boot" for the Muscular Dystrophy Association. Every dollar that motorists pitch into the firefighters' boots helps the firefighters union and its members assist Berks County families living with neuromuscular diseases get medical treatment and support services. Firefighters will be collecting at 11th and Rockland streets and Third and Washington streets from 10:30 a.m. to 5 p.m. The International Association of Fire Fighters has been a national MDA partner for more than 50 years and remains committed to the fight to end neuromuscular diseases. "We know the money we raise definitely makes a difference in the lives of people affected by neuromuscular diseases, and that it helps them get the care they need," said Mike Shoumlisky, president of IAFF Local 1803, the Reading firefighters union. "Fill the Boot is a proud tradition for us, and it's a great way for firefighters to be out in the community reminding residents that we are here, always ready to help." Continued here: Reading firefighters will hit streets to give muscular dystrophy the boot … Continue reading →

Husband of Barbour County Prosecutor Arrested for Drugging Wife Husband of Barbour County Prosecutor Arrested for Drugging Wife Updated: Tuesday, May 14 2013 10:56 AM EDT2013-05-14 14:56:40 GMT The husband of Barbour County's prosecuting attorney is free on a $5,000 personal recognizance bond after admitting that he drugged his wife in September 2012. The husband of Barbour County's prosecuting attorney is free on a $5,000 personal recognizance bond after admitting that he drugged his wife in September 2012. Updated: Tuesday, May 14 2013 10:17 AM EDT2013-05-14 14:17:22 GMT A gas explosion occurred at the Airgas plant in the Black Betsy area of Putnam County just before 3:30 p.m. on Monday. A gas explosion occurred at the Airgas plant in the Black Betsy area of Putnam County just before 3:30 p.m. on Monday. Updated: Tuesday, May 14 2013 8:14 PM EDT2013-05-15 00:14:46 GMT Huntington Prep's Andrew Wiggins signed with the University of Kansas on May 14. Huntington Prep's Andrew Wiggins signed with the University of Kansas on Tuesday in Huntington. See the rest here: Firefighters in Teays Valley to host 'Fill the Boot' drive to benefit Muscular Dystrophy Association … Continue reading →

CAMBRIDGE, MA--(Marketwired - May 9, 2013) - Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today reported financial results for the three months ended March 31, 2013, and provided an update of recent corporate developments. "We are encouraged by our initial interactions with the U.S. Food and Drug Administration regarding a potential accelerated approval regulatory path for eteplirsen for the treatment of Duchenne muscular dystrophy, and we are finalizing a response to the FDA's request for more data to help us determine the feasibility of this pathway," said Chris Garabedian, president and chief executive officer of Sarepta Therapeutics. "In parallel, we continue to advance the clinical, regulatory, and manufacturing activities for eteplirsen and our additional DMD product candidates to build a successful franchise in this important disease area." Financial Results For the first quarter of 2013, Sarepta reported a Non-GAAP operating loss of $13.3 million, compared to a Non-GAAP operating loss of $6.1 million for the first quarter of 2012. The incremental loss is primarily the result of a $6.7 million decrease in government contract revenues as well as a $0.5 million increase in operating expenses. On a GAAP basis, the operating loss for the first quarter … Continue reading →

May 7, 2013 Laminopathies are hereditary diseases that affect mainly the muscle tissue. These diseases include for example Emery-Dreifuss Muscular dystrophy, dilated cardiomyopathy, limb-girdle muscular dystrophy and Hutchison-Gilford progeria syndrome. The underlying defect in these diseases is mutation in the genes encoding lamins or lamin-associated proteins. For example, many mutations in the lamin gene LMNA have been associated with different diseases. Lamins are crucial components of the nuclear lamina that underlies the inner side of nuclear envelope, and provides mechanical stability to the nucleus. Lamina also participates in many different nuclear processes. Two theories exist, why mutations in the lamina components cause disease. According to the first theory, mutations cause changes in the nuclear structure, which can lead to cell death in tissues that undergo harsh mechanical strain, such as the muscle. The second theory postulates that disturbed lamina causes changes in the gene expression patterns that are then deleterious for the cell. A collaborative study between American and Finnish scientists bridge these two theories. The study shows that abnormal structure of the nuclear lamina, caused by laminopathy mutations, lead to changes in gene expression by disturbing the function of a specific transcription regulating protein. The researchers found out that … Continue reading →

Justin Lopez, aka DJ Ju5tin, has trouble speaking and reading. But the 8-year-old has found a way to express himself: blending beats at his mixing board. The Bethlehem boy was born with a rare form of muscular dystrophy known as glycogen storage disease. It affects his liver, muscle strength and speech. But the upcoming DJ has found his voice through music. He already has performed at Crocodile Rock Cafe and other local venues, and he will perform 3-4 p.m. June 23 at ArtsQuest's Sabor! Latin Festival at the SteelStacks campus in Bethlehem. "It keeps him busy so that he doesn't have time to think about what he has," his father Dan says. He introduced his son to electronic music when Justin was still a baby. A bank manager, Dan would come home from work and go into the basement to relax, mixing salsa, merengue and bachata songs in front of the computer. When Justin turned 4, he regularly joined his father downstairs. Dan noticed that the boy was sensitive to the beats, bouncing his head in time. "The only thing that would calm him down was music," Dan explains. At age 5, the boy asked his dad to let him … Continue reading →

Becker's muscular dystrophy stem cell treatment - rejuvaa Mohd Afzal suffering from becker's muscular dystrophy has improved after getting treated at Rejuvaa. Listen to his inspiring story from his Father. Patient h... By: Rejuvaa … Continue reading →

'Miles for Mitchell' to honor life of inspiring boy with muscular dystrophy By Devon Dolan May 3rd, 2013 @ 8:22am HERRIMAN The city of Herriman will host a fundraiser Saturday to honor the life of a young boy whose broken heart touched hearts all over the world. Mitchell Jones passed away on March 2 from heart failure after a lifelong battle with Duchenne muscular dystrophy (DMD). He was 10 years old and had been suffering from DMD since July of 2005. His father chronicled his final months in journal posts on the Facebook page Mitchell's Journey. The page now has more than 94,000 followers and Mitchell's family is using it to raise awareness and money for research and treatment. "It's still hard, but we want to make something positive out of this tragedy," said Natalie Jones, Mitchell's mother. On Saturday they are inviting the community to participate in Miles for Mitchell, a 5k race being held in memory of their son. The race starts at 9 a.m. at Herriman Main Street Park, located at 13000 South 5900 West. Registration for the event ends at 8:30 a.m. Saturday. Registered participants will also receive a t-shirt for the event and to support … Continue reading →

NEWPORT BEACH, Calif.--(BUSINESS WIRE)-- CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, will host a webinar on drisapersen with GlaxoSmithKline (GSK) on May 6, 2013 at 12 p.m. EDT for Duchenne parents and advocates. The purpose of the webinar is to update the Duchenne community about Phase II clinical trial results for GSKs drisapersen drug for the treatment of Duchenne muscular dystrophy, a progressive muscle wasting disease that impacts 1 in 3,500 boys. GSK investigator John Kraus will present and answer submitted questions. CureDuchenne is hosting the webinar in collaboration with Parent Project Muscular Dystrophy and the Muscular Dystrophy Association to enable the Duchenne community and GSK to come together to further understand the drisapersen update for potential Duchenne treatments. Boys with Duchenne are usually diagnosed before the age of 5, in a wheelchair by age 12 and most dont survive their mid-20s. There is currently no cure for Duchenne. To register for the Drisapersen Update webinar with the GSK on May 6 click here. Follow us on Facebook, Twitter and YouTube. About CureDuchenne CureDuchenne is a national nonprofit organization located in Newport Beach, Calif., dedicated to finding a cure for … Continue reading →