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(UT Southwestern Medical Center) DALLAS - Nov. 4, 2020 - A mutation in the gene that causes cystic fibrosis may accelerate heart function decline in those with Duchenne muscular dystrophy (DMD), a new study by UT Southwestern researchers suggests. The findings, published online recently in theJournal of the American Heart Association, could help doctors develop new strategies to preserve heart function in this population, potentially extending patients' lives. (Source: EurekAlert! - Medicine and Health)
Long-term treatment with ataluren delays loss of ambulation and may delay decline in pulmonary function in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD), according to study results.Medscape Medical News (Source: Medscape Neurology and Neurosurgery Headlines)
Bayer acquires Asklepios BioPharmaceutical to broaden innovation base in cell and gene therapyAcquisition fuels Bayer's cell and gene therapy platform with potential to bring urgently needed treatments to patients across multiple disease areas with high unmet need / AskBio's industry leading AAV-based gene therapy platform already yielding commercial and clinical stage assets with potential of helping larger patient populations / Portfolio includes investigational pre-clinical and clinical stage development candidates for the treatment of neuromuscular, central nervous system, cardiovascular and metabolic diseases such as therapeutics for Pompe disease, Parkinson's disease and congestive heart failure, as well as out-licensed clinical candidates for hemophilia and Duchenne muscular dystrop...
UCLA researchers have identified a compound that can reproduce the effect of exercise in muscle cells in mice. Thefindings are published in the journal Cell Reports Medicine.Normally, muscles get stronger as they are used, thanks to a series of chemical signals inside muscle cells. The newly identified compound activates those signals, which suggests that compounds like it could eventually be used to treat people with limb girdle muscular dystrophy, a form of adolescent-onset muscular dystrophy.When muscles aren ’t worked regularly, they gradually atrophy. (The phenomenon is familiar to anyone who’s had a cast on their leg for several weeks.) Fortunately, for people with healthy muscles, that deterioration is reversible. Muscle use stimulates chemical messengers inside the muscle cells...
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(Duke University Medical Center) A new drug offers hope for young boys with the progressive neuromuscular disease Duchenne muscular dystrophy (DMD) by potentially offering an alternative to high-dose glucocorticoids that have significant side effects. (Source: EurekAlert! - Medicine and Health)
(Sanford Burnham Prebys Medical Discovery Institute) Scientists at Sanford Burnham Prebys, Fondazione Santa Lucia IRCCS, and Universit à Cattolica del Sacro Cuore have shown that pharmacological (drug) correction of the content of extracellular vesicles released within dystrophic muscles can restore their ability to regenerate muscle and prevent muscle scarring. The study, published in EMBO Reports, reveals a promising new therapeutic approach for Duchenne muscular dystrophy (DMD), an incurable muscle-wasting condition, and has far-reaching implications for the field of regenerative medicine. (Source: EurekAlert! - Biology)
Researchers have identified a possible treatment for Duchenne muscular dystrophy, a rare genetic disease for which there is currently no cure or treatment. (Source: Yale Science and Health News)
Basel, 11 September 2020 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new data that show OCREVUS® (ocrelizumab) is a highly effective treatment option for people with relapsing-remitting multiple sclerosis (RRMS) who experienced a suboptimal response to their prior disease modifying therapy (DMT). Subgroup analys is from the two-year open-label Phase IIIb CASTING study also demonstrates that patients benefit across a wide range of disease related and demographic subgroups, regardless of prior treatment background. Findings will be presented at MSVirtual2020, the 8th Joint Meeting of the Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) and the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS).“For a wide range of people w...