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(University of California - Irvine) A new study, led by the University of California, Irvine (UCI), reveals how chronic inflammation promotes muscle fibrosis, which could inform the development of new therapies for patients suffering from Duchenne muscular dystrophy (DMD), a fatal muscle disease. (Source: EurekAlert! - Medicine and Health)
Basel, 16 April 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new OCREVUS ® (ocrelizumab) analyses supporting its significant benefit on disease progression in early-stage relapsing-remitting multiple sclerosis (RRMS) and primary progressive MS (PPMS) as well as demonstrating high persistence and strong adherence to twice-yearly (six-monthly) dosing. These data are being presented virtually at the 73rd American Academy of Neurology (AAN) Annual Meeting from 17–22 April 2021. OCREVUS is the number one prescribed MS medication in the U.S. for patients starting a new treatment, and more than 200,000 people have now been treated with OCREVUS globally.“All patients regardless of their form of MS experience disease progression from the start. Therefore, we are encouraged by thes...<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>
(University of Minnesota Medical School) New research from the University of Minnesota Medical School found mutations in a novel gene that may help identify patients with a specific form of muscular dystrophy. (Source: EurekAlert! - Medicine and Health)
(Critical Path Institute (C-Path)) C-Path announced today that it will open access to the Duchenne Regulatory Science Consortium database to qualified researchers, through its Rare Disease Cures Accelerator, Data and Analytics Platform. The D-RSC database includes data from Duchenne muscular dystrophy clinical trials, natural history studies and clinical data collections. The contributors of these datasets have given permission for this data to be shared externally to accelerate therapy development for DMD. (Source: EurekAlert! - Medicine and Health)
Basel, 23 April 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the European Medicines Agency ’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of ENSPRYNG® (satralizumab) as the first subcutaneous treatment option for adults and adolescents from 12 years of age living with anti-aquaporin-4 antibody (AQP4-IgG) seropositive neuromyelitis optica spe ctrum disorder (NMOSD), as a monotherapy or in combination with immunosuppressive therapy (IST). AQP4-IgG are present in around 70-80% of people with NMOSD, who tend to experience a more severe disease course.NMOSD is a rare, lifelong and debilitating autoimmune disorder of the central nervous system, often misdiagnosed as multiple sclerosis, that primarily damages the optic nerve(s) and...
(Max Delbr ü ck Center for Molecular Medicine in the Helmholtz Association) A new gene-editing technique can be used to correct mutations in muscle stem cells, paving the way for the first potential cell therapy for genetic muscle disorders. The ECRC team led by Professor Simone Spuler have published their findings in the journal JCI Insight. (Source: EurekAlert! - Medicine and Health)
Twenty-four years ago, Bobbi Heinold, 17, was diagnosed with facioscapulohumeral muscular dystrophy, a genetic muscle condition characterized by weakness, especially in the face, shoulder blades and upper arms. Doctors told her it could affect her quality of life. Bobbi and her mom drove from their Indiana home to Mayo Clinic in Rochester, Minnesota, to learn [...] (Source: News from Mayo Clinic)<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>
This poignant documentary about two young brothers with Duchenne muscular dystrophy celebrates the power of love and togethernessHere is a deeply personal documentary that raises awareness about a disability without neglecting the interiority of those living with the condition. Co-directed by Riccardo Servini and Nick Taussig, the film follows the Taussig family ’s experience of Duchenne muscular dystrophy, a genetic disorder that affects their young sons, Theo and Oscar.Duchenne is incurable and fatal. By the time Theo and Oscar are in their teens, they will be using wheelchairs full time; their 20s will come with ventilators. Throughout the film, Taussig gently explains these realities to his sons, with no sugarcoating. Satisfying children ’s curiosity has never been easy, and here t...
Basel, 28 June 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the European Commission (EC) has approved ENSPRYNG ® (satralizumab) for the treatment of adults and adolescents from 12 years of age living with anti-aquaporin-4 antibody (AQP4-IgG) seropositive neuromyelitis optica spectrum disorder (NMOSD), as a monotherapy or in combination with immunosuppressive therapy (IST). ENSPRYNG is the first and only NMO SD treatment that is administered subcutaneously every four weeks, allowing home-dosing after appropriate training.“An NMOSD relapse can be devastating, causing permanent neurological damage and disability that accumulates with subsequent relapses, which is why our goal is to prevent them,” said Prof. Dr. Friedemann Paul, Professor of Clinical Neuroimmunology, Cha...
Title: What Are the Stages of Muscular Dystrophy?Category: Diseases and ConditionsCreated: 6/17/2021 12:00:00 AMLast Editorial Review: 6/17/2021 12:00:00 AM (Source: MedicineNet Kids Health General)