Browsing Health Sciences by Subject "Precision medicine"

Now showing items 1-5 of 5

    • Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry disease and migalastat-amenable variants: data from the phase 3 randomized, multicenter, double-blind clinical trial and extension study 

      Germain, Dominique P.; Nicholls, Kathy; Giugliani, Roberto; Bichet, Daniel G.; Hughes, Derralynn A.; Barisoni, Laura; Colvin, Robert B.; Jennette, J. Charles; Skuban, Nina; Castelli, Jeffrey P.; Benjamin, Elfrida R.; Barth, Jay; Viereck, Christopher (2019) [Journal article]
      Purpose: Outcomes in patients with Fabry disease receiving migalastat during the phase 3 FACETS trial (NCT00925301) were evaluated by phenotype. Methods: Data were evaluated in two subgroups of patients with migalastat-amenable ...

    • Metabolomic biomarker candidates for skeletal muscle loss in the collagen-induced arthritis (CIA) model 

      Alabarse, Paulo Vinicius Gil; Silva, Jordana Miranda de Souza; Espírito Santo, Rafaela Cavalheiro do; Oliveira, Marianne Schrader de; Almeida, Andrelise Simões de; Oliveira, Mayara Souza de; Immig, Mônica Luiza; Freitas, Eduarda Correa; Teixeira, Vivian de Oliveira Nunes; Bathurst, Camilla L.; Brenol, Claiton Viegas; Filippin, Lidiane Isabel; Young, Stephen Peter; Lora, Priscila Schmidt; Xavier, Ricardo Machado (2021) [Journal article]
      There is no consensus for diagnosis or treatment of RA muscle loss. We aimed to investigate metabolites in arthritic mice urine as biomarkers of muscle loss. DBA1/J mice comprised collagen-induced arthritis (CIA) and control ...

    • Precision medicine for lysosomal disorders 

      Vairo, Filippo Pinto e; Málaga, Diana Elizabeth Rojas; Kubaski, Francyne; Souza, Carolina Fischinger Moura de; Poswar, Fabiano de Oliveira; Baldo, Guilherme; Giugliani, Roberto (2020) [Journal article]
      Precision medicine (PM) is an emerging approach for disease treatment and preventionthat accounts for the individual variability in the genes, environment, and lifestyle of each person.Lysosomal diseases (LDs) are a group ...

    • Precision non-implantable neuromodulation therapies : a perspective for the depressed brain 

      Borrione, Lucas; Passos, Ives Cavalcante; Salum Junior, Giovanni Abrahão; Brunoni, Andre Russowsky (2020) [Journal article]
      Current first-line treatments for major depressive disorder (MDD) include pharmacotherapy and cognitive-behavioral therapy. However, one-third of depressed patients do not achieve remission after multiple medication trials, ...

    • Toward precision medicine in ADHD 

      Buitelaar, Jan K.; Caye, Arthur; Leffa, Douglas Teixeira; Rohde, Luis Augusto Paim; Banaschewski, Tobias (2018) [Journal article]
      Attention-Deficit Hyperactivity Disorder (ADHD) is a complex and heterogeneous neurodevelopmental condition for which curative treatments are lacking. Whilst pharmacological treatments are generally effective and safe, ...