Duchenne muscular dystrophy (DMD) is a progressive muscle disorder that causes the loss of both muscle function and independence. DMD is perhaps the most prevalent of the muscular dystrophies and is the most common lethal genetic disorder diagnosed during childhood today. Each year, approximately 20,000 children worldwide are born with DMD (one of every 3,500 male children).
Cells made by fusing a normal human muscle cell with a muscle cell from a person with Duchenne muscular dystrophy --a rare but fatal form of muscular dystrophy -- were able to significantly improve muscle function when implanted into the muscles of a mouse model of the disease.
Diagnostic and treatment advances are helping patients with Duchenne muscular dystrophy-;one of nine major types of muscular dystrophy that affects males-;live into their 30s and beyond, raising challenges in such areas as education, vocation, levels of independence, personal relationships, emotional health, and intimacy.
The University of Plymouth will be continuing its research into the advancement of neuro-tumor treatments thanks to more than £100,000 from Great Ormond Street Hospital Children's Charity and Sparks, the medical research charity.
Injections of cardiac progenitor cells help reverse the fatal heart disease caused by Duchenne muscular dystrophy and also lead to improved limb strength and movement ability, a new study shows.
Scientists have developed a CRISPR gene-editing technique that can potentially correct a majority of the 3,000 mutations that cause Duchenne muscular dystrophy (DMD) by making a single cut at strategic points along the patient's DNA, according to a study from UT Southwestern Medical Center.
Researchers have successfully created artificial human muscles in the laboratories that are tiny but fully functional. These small muscles actually respond to the nerve stimulations, says the latest study reporting this scientific breakthrough.
The commissioner of the Food and Drug Administration questions whether the right financial incentives are in place for drugmakers who develop orphan drugs for rare diseases.
Researchers at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA have discovered how high glucose levels -- whether caused by diabetes or other factors -- keep heart cells from maturing normally.
In a paper published in the Nature journal Scientific Reports, Saint Louis University researchers report that a new drug reduces fibrosis (scarring) and prevents loss of muscle function in an animal model of Duchenne muscular dystrophy (DMD), providing a promising approach in designing new medications for those suffering from DMD.
Regenerative medicine researchers at UT Southwestern Medical Center developed an improved and simplified gene-editing technique using CRISPR/Cas9 tools to correct a common mutation that causes Duchenne muscular dystrophy.
A unique initiative launched today by muscular dystrophy charity Duchenne UK is set to radically simplify the way necessary evidence is generated for submissions made to health technology appraisals bodies such as the National Institute for Health and Care Excellence and the Haute Autorité de Santé.
After boys and young men with Duchenne muscular dystrophy received cardiac progenitor cell infusions, medical tests indicated that the patients' hearts appeared improved, results from a new study show. Patients in the study also scored higher on arm strength tests after receiving the cell infusions.
A new study from UT Southwestern suggests that more people with Duchenne muscular dystrophy could live longer by identifying and more aggressively treating patients with certain risk factors.
Scientists at the University of California, Berkeley, have engineered a new way to deliver CRISPR-Cas9 gene-editing technology inside cells and have demonstrated in mice that the technology can repair the mutation that causes Duchenne muscular dystrophy, a severe muscle-wasting disease.
A new study published in the journal Nature Communications describes a breakthrough in research related to facioscapulohumeral muscular dystrophy (FSHD). The debilitating genetic disease - which has no approved treatment - affects an estimated 38,000 Americans and causes degeneration and wasting of the skeletal muscles.
Researchers from the Perelman School of Medicine at the University of Pennsylvania have made a discovery about muscular dystrophy disorders that suggest new possibilities for treatment.
A new study from the Cedars-Sinai Heart Institute, Los Angeles, published by the European Heart Journal, forecasts the use of infusions of cardiac stem cells obtained from young hearts in reversing the process of aging in the human heart.
Cardiac stem cell infusions could someday help reverse the aging process in the human heart, making older ones behave younger, according to a new study from the Cedars-Sinai Heart Institute.
"Good morning, doctor, I am here for my gene editing appointment." In the future, could this be a greeting heard in physician offices around the world? With the introduction of CRISPR technology, genetic material can now be more easily and precisely edited, even creating changes that can subsequently be inherited by offspring.
Researchers from Genethon, the AFM-Telethon laboratory, Inserm (UMR 1089, Nantes) and the University of London (Royal Holloway) demonstrated the efficacy of an innovative gene therapy in the treatment of Duchenne muscular dystrophy.