Category Archives: Muscular Dystrophy Treatment

Thanks to advances in many areas of medicine, such as cardiology and pulmonology, people with Duchenne muscular dystrophy in the 21st century are living longer than in previous decades, often well into adulthood. The use of available treatments can help maintain comfort and function and prolong life. Talk with anMDA clinicphysician for more information. People with DMD may haveunexpected adverse reactions to certain types of anesthesia. It's important that the surgical team know about the patient's DMD so that complications can be avoided or quickly treated. Braces, also calledorthoses, support the ankle and foot, or may extend up over the knee.Ankle-foot orthoses (AFOs) are sometimes prescribed for night wear to keep the foot from pointing downward and keep the Achilles tendon stretched while the child is sleeping. Standing for a few hours each day, even with minimal weight bearing, promotes better circulation, healthier bones and a straight spine. Astanding walker or standing framecan assist people with DMD to stand. Some wheelchairs will raise the user into a standing position. Sooner or later, a wheelchair is needed in DMD, typically by about age 12. Unless there's an injury, such as a broken leg, wheelchair use usually is gradual. Many at first … Continue reading →

Muscular dystrophy (MD) is a group of hereditary muscle diseases. MD results in weakening the skeletal muscles of body. The main traits of muscular dystrophy are progressive skeletal muscle weakness, defects in muscle proteins, and death of muscle cells and tissue. Muscular dystrophy has many sub-types, and most are multiple-system disorders, presenting as manifestation in body systems, such as heart, nervous system, endocrine system, gastrointestinal system, eyes, etc. Thus, many signs and symptoms may occur, including: Progressive muscular wasting; Muscle weakness; Calf deformation; Drooping eyelids; Poor balance; Progressive crippling; Walking difficulty; Frequent falls; Wadding gait; Respiratory difficulty; Scoliosis; Incontinence, etc. Our stem cell therapy for muscular dystrophy can potentially bring improvements such as The stem cell treatment can slow down the loss of muscle mass and relieve the symptoms. However it is important to remember that the treatment is not a cure and it cannot eliminate the underlying cause of muscle fiber loss. Thus, improvements are not permanent and will last for a certain period of time depending on how fast the disease is progressing. Upon receiving your medical information our doctors can give you additional guidance regarding your/your relatives particular condition. Read more: Alternative Treatment for Muscular Dystrophy | … Continue reading →

Gene therapy, the use of genetic material to treat a disease or disorder, is making strides in muscular dystrophy. Although the approach is still considered experimental, studies in animal models have shown promising results and clinical trials in humans are underway. Gene therapy has the potential to help people with inherited disorders, in which a gene mutation causes cells to produce a defective protein or no protein at all, leading to disease symptoms. To deliver the genetic material to the cells, scientists use a tool called a vector. This is typically a virus that has been modified so that it doesnt cause disease. It is hoped that the vector will carry the therapeutic gene into the cells nucleus, where it will provide the instructions necessary to make the desired protein. The most common form of muscular dystrophy, Duchenne muscular dystrophy, is caused by a mutation in the DMD gene, which codes for a protein called dystrophin. Dystrophin is part of a protein complex that strengthens and protects muscle fibers. When the cells dont have functional dystrophin due to the gene mutation, muscles progressively weaken. Scientists think that supplying a gene that codes for a functional form of dystrophin might be … Continue reading →

March 15, 2018March 15, 2018 News TheU.S. Food and Drug Administration has placed a clinical hold on a Phase 1/2 trial testingSGT-001, a dystrophin gene therapy intended to treat Duchenne muscular dystrophy (DMD), after the ... Read more March 14, 2018 News Sarepta Therapeuticswill seek accelerated U.S. approval of a treatment for a subset ofDuchenne muscular dystrophy (DMD) patients. Golodirsen (SRP-4053)is for patients whose disease stems from a deleted part of ... Read more March 14, 2018 News When it comes to rare diseases, one that definitely makes the list is spinal muscular atrophy with respiratory distress SMARD, for short. Hunter Pageau, a 12-year-old boy from ... Read more March 13, 2018March 12, 2018 columns, You, Me and MD - a Column by Leah Leilani Being wheelchair-bound and still stylish can be the ultimate challenge. Its hard to find that balance between style and comfort, especially as a woman. The average, able-bodied female millennial ... Read more March 12, 2018 News In his 10 months on the job, Commissioner Scott Gottlieb of the U.S. Food and Drug Administration is earning praise for his efforts to make clinical trials for new ... Read more March 12, 2018March 12, 2018 News Research … Continue reading →

Fred Hutch biologist Dr. Stephen Tapscott. (Fred Hutch Photo) A new study conducted at the Fred Hutchinson Cancer Research Center in Seattle has found a new potential target for treating or even reversing facioscapulohumeralmuscular dystrophy, a rare but devastating disease that currently has no cure. The disease is caused by theDUX4 gene, which is normally shut off very early in a fetus development. If someone is born with too few copies of the gene, it will spontaneously turn back on, leading to a breakdown in muscle cells that eventually leads to difficulty with basic movements like walking, speaking and swallowing. Researchers at Fred Hutch used a proteomics approach the relatively young field of studying proteins along with CRISPR gene targeting to identify two groups of proteins responsible for turning the gene off in both stem and muscle cells. Using that knowledge, scientists could theoretically create a treatment that prevents or reverses the disease by shutting down the DUX4 gene.The study was led by Fred Hutch biologistDr. Stephen Tapscottand staff scientist Dr. Amy Campbell and was published Tuesday in scientific journal eLife. While such a treatment would still be years down the line, the results are an important step toward a … Continue reading →

The different types of muscular dystrophy affect different sets of muscles and cause different degrees of muscle weakness. Duchenne muscular dystrophy is the most common and the most severe form of MD. It affects about 1 out of every 3,500 boys. (Girls can carry the gene that causes the disease, but they usually have no symptoms.) This form occurs because of a problem with the gene that makes dystrophin. Without this protein, the muscles break down and a child becomes weaker. Children with Duchenne MD usually begin to have problems around age 5, as the pelvic muscles begin to weaken. Most kids with this form need to use a wheelchair by age 12. Over time, their muscles weaken in the shoulders, back, arms, and legs. Eventually, the respiratory (breathing) muscles and heart muscle also may weaken in the teenage years, requiring further treatments. Although most kids with Duchenne MD have average intelligence, about a third experience learning disabilities and a small number are intellectually disabled. Advances in medical care are helping kids with Duchenne to live longer and healthier lives. While the incidence of Duchenne is known, it's unclear how common other forms of MD are because the symptoms can … Continue reading →

The different types of muscular dystrophy affect different sets of muscles and cause different degrees of muscle weakness. Duchenne muscular dystrophy is the most common and the most severe form of MD. It affects about 1 out of every 3,500 boys. (Girls can carry the gene that causes the disease, but they usually have no symptoms.) This form occurs because of a problem with the gene that makes dystrophin. Without this protein, the muscles break down and a child becomes weaker. Children with Duchenne MD usually begin to have problems around age 5, as the pelvic muscles begin to weaken. Most kids with this form need to use a wheelchair by age 12. Over time, their muscles weaken in the shoulders, back, arms, and legs. Eventually, the respiratory (breathing) muscles and heart muscle also may weaken in the teenage years, requiring further treatments. Although most kids with Duchenne MD have average intelligence, about a third experience learning disabilities and a small number are intellectually disabled. Advances in medical care are helping kids with Duchenne to live longer and healthier lives. While the incidence of Duchenne is known, it's unclear how common other forms of MD are because the symptoms can … Continue reading →

Some Patients Give Up .. Some others Dont Give Up... and for Some Others , we don't let them to Give Up Even A Slight delay of the Progress of the Disease Gains Valuable time for our Lives untill the Final Cure . (M.D & M.S Members of Royal Cells Group ) --------------------------- The Great Importance of delaying the Progress of : Muscular Dystrophy & Multiple Sclerosis Muscular Dystrophy & Multiple Sclerosis belong to Degenerative Incurable Diseases today that affect progressively and other Vital organs of the Human Organism with great risk for severe complications . Until the Day that the Final Cure will be invented , Patients must Follow Natural Remedies that are enabled to Special Nutrition & Special Physiotherapy , that are able to push back & delay the maximum possible the progress of these diseases. Royal Cells Group contributes to this effort , to Gain the extra time Patients need, in order to stand firm till the cure of these diseases will be discovered in future . Royal Cells Group Is a Group of patients - all around the world - who use voluntarily Nectar "T Natural Daily Nutrition additionally with Special food in order to: *Achieve … Continue reading →

What are Duchenne and Becker muscular dystrophy? Muscular dystrophy is where the muscles weaken and lose muscle mass; in this case, both Duchenne and Becker muscular dystrophy are caused by a genetic mutation in the dystrophin gene. Subscribe - https://goo.gl/w5aaaV. More videos - https://goo.gl/UhOKiM. Support us on Patreon - https://goo.gl/ZGHEk4. This video covers the pathophysiology of both, as well as clinical signs and symptoms, and diagnosis, and management. Subscribe - http://www.youtube.com/channel/UCNI0q... This video is brought to you by Osmosis. Along with providing open-access videos, Osmosis offers a comprehensive e-learning platform that connects med students with thousands of flashcards and quiz questions, depending on each student's needs. Ever wish information would just diffuse into your brain? Well, Osmosis helps make that possibledon't learn it, osmose it! https://www.osmosis.org/ Support us on Patreon! - https://goo.gl/izRx2z We also have free practice questions for the USMLE and NCLEX-RN exams here: https://goo.gl/3oGOEi Also, we're social: Facebook - https://www.facebook.com/OsmoseIt/Twitter - https://twitter.com/osmoseit Got feedback? We'd love to hear it! http://goo.gl/forms/T6de48NVzR This video is licensed under a Creative Commons CC-BY-SA 4.0 international license, which means that you're free to share and adapt it so long as you follow the Attribution and ShareAlike terms and conditions! Our supporters:Wade Licup Sources:Sarkozy … Continue reading →

Duchenne muscular dystrophy (DMD) is a severe type of muscular dystrophy.[2] The symptom of muscle weakness usually begins around the age of four in boys and worsens quickly.[1] Typically muscle loss occurs first in the upper legs and pelvis followed by those of the upper arms.[2] This can result in trouble standing up.[2] Most are unable to walk by the age of 12.[1] Affected muscles may look larger due to increased fat content.[2]Scoliosis is also common.[2] Some may have intellectual disability.[2] Females with a single copy of the defective gene may show mild symptoms.[2] The disorder is X-linked recessive.[2] About two thirds of cases are inherited from a person's parents, while one third of cases are due to a new mutation.[2] It is caused by a mutation in the gene for the protein dystrophin.[2] Dystrophin is important to maintain the muscle fiber's cell membrane.[2]Genetic testing can often make the diagnosis at birth.[2] Those affected also have a high level of creatine kinase in their blood.[2] No cure for muscular dystrophy is known.[1]Physical therapy, braces, and corrective surgery may help with some symptoms.[1]Assisted ventilation may be required in those with weakness of breathing muscles.[2] Medications used include steroids to slow muscle … Continue reading →