What is the level of dystrophin expression required for effective therapy of Duchenne muscular dystrophy?
Sign Up to like & getrecommendations! Published in 2019 at "Journal of Muscle Research and Cell Motility"
DOI: 10.1007/s10974-019-09535-9
Abstract: Duchenne muscular dystrophy (DMD) is a fatal X-linked muscle wasting disease. The disease is due to mutations in the DMD gene that encodes for a large intracellular protein called dystrophin. Dystrophin plays a critical role… read more here.
Keywords: effective therapy; muscle; muscular dystrophy; expression required ... See more keywords
Impact of a Laboratory-Developed Phenotypic Rapid Susceptibility Test Directly From Positive Blood Cultures on Time to Narrowest Effective Therapy in Patients With Gram-Negative Bacteremia: A Prospective Randomized Trial
Sign Up to like & getrecommendations! Published in 2022 at "Open Forum Infectious Diseases"
DOI: 10.1093/ofid/ofac347
Abstract: Abstract Background Antimicrobial susceptibility testing (AST) is often needed prior to antimicrobial optimization for patients with gram-negative bloodstream infections (GN-BSIs). Rapid AST (rAST) in combination with antimicrobial stewardship (AS) may decrease time to administration of… read more here.
Keywords: effective therapy; time; therapy; narrowest effective ... See more keywords
CD19-CAR-T cells are an effective therapy of post-transplant relapse in B- ALL patients: Real-World Data from Germany.
Sign Up to like & getrecommendations! Published in 2023 at "Blood advances"
DOI: 10.1182/bloodadvances.2022008981
Abstract: Key Points • Most patients with refractory B-ALL could achieve remission with a single infusion of CAR T cells.• Survival probability is excellent for patients who relapsed >6 months posttransplant and poor for patients who… read more here.
Keywords: effective therapy; cd19 car; car; cells effective ... See more keywords
The Search for an Effective Therapy to Treat Fragile X Syndrome: Dream or Reality?
Sign Up to like & getrecommendations! Published in 2017 at "Frontiers in Synaptic Neuroscience"
DOI: 10.3389/fnsyn.2017.00015
Abstract: Fragile X Syndrome (FXS) is the most common form of intellectual disability and a primary cause of autism. It originates from the lack of the Fragile X Mental Retardation Protein (FMRP), which is an RNA-binding… read more here.
Keywords: search effective; effective therapy; fxs; fragile syndrome ... See more keywords