Category Archives: Muscular Dystrophy Treatment

From left, Sage Therapeutics Amy Schacterle, Al Robichaud, chief executive Jeff Jonas, Chris Silber, and Frank Salituro posed for a portrait at their Cambridge office. Earlier in his career, Paul Bolno worked with neuroscientists at drug giant GlaxoSmithKline PLC. Today, as chief executive of Wave Life Sciences Ltd., he is leading the Cambridge biotechs effort to advance two experimental drugs that could become the first treatments for the progressive brain disorder Huntingtons disease. Bolnos move from Big Pharma to biotech reflects a broader transition at businesses working on medicines for neurological diseases. As more than 10,000 doctors and scientists converge on Boston this week for the annual convention of the American Academy of Neurology, much of the fields progress is being made inside smaller entrepreneurial companies. Advertisement The biotechs are moving the science forward, said Bolno, whose company last week closed on a $150 million financing round and will open a Lexington plant this summer to make drugs for clinical trials. There was a mass exodus of pharma companies from the neurological space, and that created an opportunity for companies like ours. The large companies are now on the sidelines trying to get back in. With new neurodegenerative treatments finally … Continue reading →

Financial terms of the acquisition include a total upfront consideration of $140 million paid to Marathon Pharmaceuticals. Marathon is also entitled to receive payments from PTC based on annual net sales of Emflaza beginning in 2018, which PTC expects will range as a percentage of net sales between the low to mid-20s on a blended average basis. In addition, Marathon has the opportunity to receive a single $50 million sales-based milestone. About Duchenne Muscular Dystrophy Primarily affecting males, Duchenne muscular dystrophy (DMD) is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-twenties due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of skeletal, diaphragm, and heart muscles. Patients with DMD can lose the ability to walk as early as age ten, followed by loss of the use of their arms. DMD patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and twenties. More information regarding DMD is available through the Muscular Dystrophy Association and the Parent Project Muscular … Continue reading →

Joseph Penrod is 14, and has gone from walking, to using a scooter, to needing a motorized wheelchair. He has Duchenne muscular dystrophy, which is stealing his mobility. Eventually, the disease will take his life. Detroit Free Press Livonia Community Swim Club swimmer Caitlin Jodway, 16, of Livonia Franklin High School poses for a photo with Joseph Penrod, 14, at the Livonia Recreation Center in Livonia on Wednesday April 19, 2016.(Photo: Ryan Garza, Detroit Free Press)Buy Photo Caitlin Jodway's muscles flex and relax, flex and relax in a steady rhythm as herlimbs slicethe water. She glides through the pool, putting those sinewy arms and legs to work to help a boy whose own muscles are failing. That boy is Joseph Penrod. He is 14, and has gone from walking, to using a scooter, to needing a motorized wheelchair.He has Duchenne muscular dystrophy, whichis stealing his mobility. Eventually, the disease willtake his life. Autoplay Show Thumbnails Show Captions But in the time he has here on this earth, Joseph wants to be able to do the things other kids his age do, like go to the movies and hang out at the mall with his friends from Walnut Creek Middle School … Continue reading →

Collecting monetary donations to support the MDA is only half of why the Livermore Womans Club's 5K Walk/Run race exists. And on Saturday, the 5th annual 5K Walk/Run will again take place at 8 a.m. at the pavilion on Main Street on the riverfront. The other half is fueled by 20-year-old Tessa Wilkerson of Livermore. She is the inspiration of the race. McLean County has been Tessa's home for all of her life. She's also had to live just as long with spinal muscular atrophy type 2 ever since she was first diagnosed with it at 18 months old. According to the National Institutes of Health, MSA type 2 is an inherited condition marked by progressively weakening muscles in children aged 6 to 12 months old that allows them to only sit without assistance. While she's never let her condition define her, she's had to face some challenges that have come with it. "The hardest time I had was when I was between the ages of like, probably 10 and 12. I had some major surgeries done then, and I didn't go to kindergarten until late. So those times were kind of hard, but it's kind of gotten easier the … Continue reading →

New York, NY (SBWIRE) 04/14/2017 Duchenne muscular dystrophy (DMD) is one of the most common types of muscular dystrophy, which is an X-linked inherited genetic disorder characterized by gradual skeletal muscle weakness. According to NHS, in every year 100 boys are born with Duchenne muscular dystrophy in the U.K. As per DMD surveillance report by Center for Disease Control and Prevention (CDC), 349 new cases of Duchenne muscular dystrophy were reported in 2010 in the U.S. Duchenne muscular dystrophy usually affects male child and first signs and symptoms of the disease are observed at the age of 2-3 years. Duchenne muscular dystrophy worsens quickly which results in loss of ability to walk and eventually causes death within 20-30 years of age. Currently there is no curative treatment for Duchenne muscular dystrophy however symptomatic treatment approaches such as pain alleviating drugs and steroids to slow down the progression of disease are recommended by majority of physicians. Treatment is mainly aimed to maximize the quality of life. Continuous research on gene therapy, exon skipping drugs and stem cell therapy for the treatment of Duchenne muscular dystrophy is expected to fuel the global market for Duchenne muscular dystrophy treatment market over the forecast … Continue reading →

Science Daily Gene-editing alternative corrects Duchenne muscular dystrophy ... Science Daily Using the new gene-editing enzyme CRISPR-Cpf1, researchers have successfully corrected Duchenne muscular dystrophy in human cells and mice in the lab. and more » Continue reading here: Gene-editing alternative corrects Duchenne muscular dystrophy ... - Science Daily … Continue reading →

Newswise BALTIMORE, MD (April 12, 2017) Three grassroots foundations founded by the families of young boys diagnosed with Duchenne muscular dystrophy (DMD) have jointly awarded a $100,000 grant to Kennedy Krieger Institutes Center for Genetic Muscle Disorders for research into novel drug therapies. The funding from Ryans Quest, of New Jersey, along with Michaels Cause and Pietros Fight, both of New York, will enable the scientific team to study existing drugs that have shown early therapeutic promise in cell cultures. "We remain dedicated to helping identify existing FDA-approved drugs that can be repurposed to help treat Duchenne muscular dystrophy and ultimately expedite potential therapies for patients," said David Schultz, co-founder of Ryans Quest and father of 11-year-old Ryan, who has DMD. Researcher Dr. Kathryn Wagner, and her colleagues Gabsang Lee and Congshan Sun, recently screened existing drugs using human myoblasts derived from induced pluripotent stem cells (iPSCs) from individuals living with DMD. The iPSCs are a type of stem cell that can be generated from mature living cells. Several drugs reliably produced a positive therapeutic response in the cells in culture. With this funding, the findings will be validated and trialed in the mouse model for DMD. As many of … Continue reading →

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CONCORD Concord firefighters are showing their support for the Muscular Dystrophy Association (MDA) as they kick off the annual Fill the Boot fundraising campaign to help kids and adults with muscular dystrophy, ALS and related muscle-debilitating diseases live longer and grow stronger. Continuing an annual tradition, dedicated Concord firefighters will hit the streets or storefronts with boots in hand asking pedestrians, motorists, customers and other passersby to make a donation to MDA on April 15, May 13 and June 10. We are thrilled to be working with Concord firefighters for another year of Fill the Boot to help provide the funds needed to find treatments and cures for muscular dystrophy, ALS and related diseases that severely limit strength and mobility, said Lisa Swicegood, Associate Executive Director, MDA Charlotte. The dedication of these firefighters to MDAs mission is unwavering, spending countless hours both with Fill the Boot and MDA Summer Camp to care for local kids and adults. In 2016, Concord firefighters FTB campaign was the second largest in the state of North Carolina and we know that their devotion to our families will make this years drive another huge success. Funds raised through Concords Fill the Boot events in 2017 … Continue reading →

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