The U.S. Food and Drug Administration has approved Capricor Therapeutics plan for further development of its CAP-1002 stem cell therapy for Duchenne muscular dystrophy, paving the way for another Phase 2 clinical trial of the treatment this year. The thumbs-up came when Capricor received official minutes from a meeting with the FDAthatincluded the companys plan to file an investigational new drug application for CAP-1002. The treatment involvesrepeated intravenous injections of CAP-1002, which consists of heart stem cells that researchers believe can help regulate immune responses. The FDAs response to our proposed clinical development plan supports our near-term objective of submitting an IND [investigational new drug application] for intravenous CAP-1002 as well as provides us with clarity on a path to potential product registration, Dr. Linda Marbn, president and CEO of Capricor, said in a press release. We look forward to commencing a randomized, double-blind, placebo-controlled Phase 2 clinical trial of intravenous, repeat-dose CAP-1002 in boys and young men with DMD in the second half of 2017, she added. Capricor is already assessing the therapy in a Phase 1/2 trial (NCT02485938) of Duchenne patients with damaged heart muscles, also known as cardiomyopathy. But thestudy is delivering the cells directly to patients … Continue reading →

The effects of a debilitating illness such as muscular dystrophy are inevitable, especially when the disease is incurable, as is the case with Duchenne muscular dystrophy. Some treatments can help to manage the condition, but this muscle-wasting disease wreaks havoc on the human body, leaving it weak and incapable of performing basic functions as the condition worsens. The possibility of stopping the effects has been explored, but being able to reverse the condition and restore strength to the muscles has always been deemed somewhat of an impossibility. However, a new hope arises in the form of gene editing. In canine trials, muscular dystrophy was completely cured and reversed, allowing the dogs to go symptom-free for the entirety of the study. In a report published in Nature Communications, a team of international researchers explored the possibility of gene therapy in dogs to reverse and ultimately cure Duchenne muscular dystrophy. The group of scientists, including Geneticist Darren Griffin of the University of Kent, used just a single injection in their study, finding an extraordinary new gene therapy. This new type of therapy targets the dystrophin gene, injecting it with microdystrophin, a compressed version of the gene itself. The dystrophin gene is relatively … Continue reading →

Muscular dystrophy (MD)is a neuromuscular disorder, characterised mainly by progressive muscle loss. Physical therapy plays an important role in MDtreatment, however there are many myths out there about the practice and its benefits. The American Physiotherapy Association has useful information to set some of these myths straight. You need a referral to be able to see a physical therapist. Contrary to popular belief, you do not need a referral to be evaluated by a physical therapist. However, some states have restrictions on the treatment a physical therapist can provide without a referral. Its painful. Many people avoid visiting a physical therapist because theyre worried its going to be painful. The objective of physical therapy is to reduce a patients pain, and the therapist works within the patients pain threshold to do so. Its only for accidents and injuries. Physical therapists are also skilled at anticipating problems before they worsen, they dont just manipulate and stretch injured muscles. MORE:Talking to family members about your childs neuromuscular disorder. The rest is here: 7 Myths About Physical Therapy - Muscular Dystrophy News - Muscular Dystrophy News … Continue reading →

Duchenne muscular dystrophy (DMD) is the most common and, sadly, the most severe form of the debilitating genetic disorder. Now researchers have used a new gene therapy technique to restore muscle strength and stabilize the symptoms of the disorder in dogs, in an important step that could one day lead to the treatment being applied to children. Affecting one in 5,000 boys (but very rarely occurring in girls), DMD usually begins to show itself around the age of three to five, and progresses quickly from there. It disrupts dystrophin, a protein that's responsible for maintaining muscle integrity and strength. As a result, DMD causes the muscles to weaken and waste away, often rendering a patient unable to walk by age 12 and unlikely to live beyond their mid-20s. UPGRADE TO NEW ATLAS PLUS More than 1,500 New Atlas Plus subscribers directly support our journalism, and get access to our premium ad-free site and email newsletter. Join them for just US$19 a year. To combat genetic disorders, researchers often insert a healthy copy of the affected gene into a viral vector essentially, a harmless shell of a virus that carries the material into cells. Unfortunately, that tactic doesn't work with DMD: … Continue reading →

The City of Myrtle Beach Fire Departmentis showing its support for the Muscular Dystrophy Association (MDA) as its members 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 a more than 60 year tradition, dedicated fire fighters from the Carolina Beach Fire Departmentwill hit the streets or storefronts with boots in hand asking pedestrians, motorists, customers and other passersby to make a donation to MDA from August 4th-6thin front of the Lowes Food on 970 Cipriana Dr. from 11 AM-5 PM, Walmart Neighborhood Markets located on Seaboard Street and 3915 N Kings Hwy and in front of the Coastal Grand mall entrances from 10 AM-4 PM.We are thrilled to be working with the Myrtle Beach Fire Department 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 Fundraising Coordinator Angelica Hickey. The dedication of these fire fighters to MDAs mission is unwavering, spending countless hours both with Fill the Boot and MDA Summer Camp to care for the kids and … Continue reading →