Itzuli orri hau
(Yale University) Researchers at Yale have identified a possible treatment for Duchenne muscular dystrophy (DMD), a rare genetic disease for which there is currently no cure or treatment, by targeting an enzyme that had been considered " undruggable. " The finding appears in the Aug. 25 edition of Science Signaling. (Source: EurekAlert! - Medicine and Health)
Basel, 10 September 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) will present new ENSPRYNG ® (satralizumab) data on reducing relapse severity in the treatment of neuromyelitis optica spectrum disorder (NMOSD), a rare disease of the central nervous system. These data are being presented at MSVirtual2020, the 8th joint ACTRIMS-ECTRIMS meeting, in addition to longer-term efficacy data supporting the continued effect of ENSPRYNG on reducing the risk of NMOSD relapse, as well as its favourable benefit:risk profile.“The data for ENSPRYNG at MSVirtual2020 are promising and suggest it significantly reduces relapse severity and frequency, which are important goals of the treatment for people with NMOSD,” said Professor Anthony Traboulsee, M.D., Neurologist and Professor, University of British Col...
Basel, 9 September 2020 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced the initiation of an innovative Phase III clinical trial programme for its investigational medicine fenebrutinib in multiple sclerosis (MS), along with a higher-dose Phase III clinical trial programme for OCREVUS® (ocrelizumab) and a distinct O CREVUS trial specifically to support African-American and Hispanic- and Latinx-American patients with MS. Overviews of clinical trials and scientific rationale will be presented at MSVirtual2020, the 8th Joint Meeting of the Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIM S) and the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) from 11-13 September 2020.“We remain committed to advancing the science in MS by in...
This review highlights recent advances in gene-specific therapies for neuromuscular diseases of childhood, including spinal muscular atrophy, Duchenne muscular dystrophy, and centronuclear myopathy.Seminars in Neurology (Source: Medscape Today Headlines)
(University of Bonn) Duchenne muscular dystrophy (DMD) is the most common muscle disease in children and is passed on by X-linked recessive inheritance. Characteristic is a progressive muscular atrophy. The disease often results in death before the third decade of life. Researchers of the Universities of Maynooth (Ireland) and Bonn have found a connection between dystrophic muscles and the lymphatic system in mice with Duchenne disease. The results have now been published in the journal " iScience " . (Source: EurekAlert! - Medicine and Health)
CAMBRIDGE, Mass., Aug. 25, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the U.S. Food and Drug Administration (FDA) has accepted the Company’s... (Source: Drugs.com - New Drug Applications)
VILTEPSO is a drug for the treatment of a particular type of Duchenne muscular dystrophy (DMD). (Source: FDA Center for Drug Evaluation and Research - What's New)<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>
THURSDAY, Aug. 13, 2020 -- Accelerated approval has been granted to Viltepso (viltolarsen) injection for treatment of patients with Duchenne muscular dystrophy (DMD) and a mutation of the DMD gene amenable to exon 53 skipping, the U.S. Food and Drug... (Source: Drugs.com - Daily MedNews)
Viltolarsen is for patients with Duchenne muscular dystrophy with a confirmed mutation amenable to exon 53 skipping.FDA Approvals (Source: Medscape Neurology and Neurosurgery Headlines)
PARAMUS, N.J., Aug. 12, 2020 /PRNewswire/ -- NS Pharma, Inc. announced today that the U.S. Food& Drug Administration (FDA) has approved Viltepso (viltolarsen) injection for patients with Duchenne muscular dystrophy (DMD) who are amenable to... (Source: Drugs.com - New Drug Approvals)