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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...<div id="medworm"><p><b><i>MedWorm Message:</i></b> Have you tried our new <a href="https://medworm.com/">medical search engine</a>? More powerful than before. Log on with your social media account. 100% free.</p></div>
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...
Dear Investor, Please find attached a press release from our partner Sarepta Therapeutics:https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-reports-sustained-functional-improvement Do not hesitate to contact us for any further questions. With best regards, (Source: Roche Investor Update)
SOUTH PLAINFIELD, N.J., Dec. 4, 2020 -- (Healthcare Sales & Marketing Network) -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Translarna (ataluren) has been granted marketing approval in the Russian Federation for nonsense mutation Duchenn... Biopharmaceuticals, Regulatory PTC Therapeutics, Translarna, ataluren, Duchenne muscular dystrophy (Source: HSMN NewsFeed)
Acquisition 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 dystrophy / Acquisition secures additionally revenue generating Contract Development and ...
(Max Delbr ü ck Center for Molecular Medicine in the Helmholtz Association) A muscle fiber consists of just one cell, but many nuclei. A team at the MDC led by Professor Carmen Birchmeier has now shown just how varied these nuclei are. The study, which has been published in Nature Communications, can help us better understand muscle diseases such as Duchenne muscular dystrophy. (Source: EurekAlert! - Medicine and Health)<div id="medworm"><p><b><i>MedWorm Message:</i></b> If you are looking to buy something in the <a href="http://thejanuarysales.com/">January Sales</a> please visit <a href="http://thejanuarysales.com/">TheJanuarySales.com</a> for a directory of all the best sales in the UK. Any income gained via affiliate links keeps MedWorm running.</p></div>
Basel, 14 December 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the U.S. Food and Drug Administration (FDA) has approved a shorter two-hour infusion time for OCREVUS ® (ocrelizumab), dosed twice-yearly for those living with relapsing or primary progressive multiple sclerosis (MS) who have not experienced any prior serious infusion reactions (IRs). The approval was based on data from the randomised, double-blind ENSEMBLE PLUS study.“More than 170,000 people with MS have been treated with OCREVUS - the only approved B-cell therapy with a twice-yearly dosing schedule - and it is the most prescribed MS medicine in the U.S.,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Developmen t. “We constantly strive to improve the exp...