Cargando…

Rimeporide as a ﬁrst- in-class NHE-1 inhibitor: Results of a phase Ib trial in young patients with Duchenne Muscular Dystrophy

Rimeporide, a ﬁrst-in-class sodium/proton exchanger Type 1 inhibitor (NHE-1 inhibitor) is repositioned by EspeRare for patients with Duchenne Muscular Dystrophy (DMD). Historically, NHE-1 inhibitors were developed for cardiac therapeutic interventions. There is considerable overlap in the pathophysi...

Descripción completa

Detalles Bibliográficos
Autores principales:	Previtali, Stefano C., Gidaro, Teresa, Díaz-Manera, Jordi, Zambon, Alberto, Carnesecchi, Stephanie, Roux-Lombard, Pascale, Spitali, Pietro, Signorelli, Mirko, Szigyarto, Cristina Al-Khalili, Johansson, Camilla, Gray, Julian, Labolle, Delphine, Porte Thomé, Florence, Pitchforth, Jacqueline, Domingos, Joana, Muntoni, Francesco
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2020
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7482441/ https://www.ncbi.nlm.nih.gov/pubmed/32535224 http://dx.doi.org/10.1016/j.phrs.2020.104999

Ejemplares similares

Biomarkers of Duchenne muscular dystrophy: current findings
por: Szigyarto, Cristina Al-Khalili, et al.
Publicado: (2018)

Blood-derived biomarkers correlate with clinical progression in Duchenne muscular dystrophy
por: Strandberg, Kristin, et al.
Publicado: (2020)

Longitudinal serum biomarker screening identifies malate dehydrogenase 2 as candidate prognostic biomarker for Duchenne muscular dystrophy
por: Signorelli, Mirko, et al.
Publicado: (2019)

Penalized regression calibration: A method for the prediction of survival outcomes using complex longitudinal and high‐dimensional data
por: Signorelli, Mirko, et al.
Publicado: (2021)

Orthogonal proteomics methods warrant the development of Duchenne muscular dystrophy biomarkers
por: Johansson, Camilla, et al.
Publicado: (2023)

Tracking disease progression non‐invasively in Duchenne and Becker muscular dystrophies
por: Spitali, Pietro, et al.
Publicado: (2018)

Circulating Biomarkers for Duchenne Muscular Dystrophy
por: Aartsma-Rus, Annemieke, et al.
Publicado: (2015)

Evaluation of serum MMP-9 as predictive biomarker for antisense therapy in Duchenne
por: Lourbakos, A., et al.
Publicado: (2017)

Longitudinal metabolomic analysis of plasma enables modeling disease progression in Duchenne muscular dystrophy mouse models
por: Tsonaka, Roula, et al.
Publicado: (2020)

Peak functional ability and age at loss of ambulation in Duchenne muscular dystrophy
por: Zambon, Alberto A., et al.
Publicado: (2022)

Genes for endosomal NHE6 and NHE9 are misregulated in autism brains
por: Schwede, M, et al.
Publicado: (2014)

The mammalian Na+/H+ antiporters NHE-1, NHE-2, and NHE-3 are electroneutral and voltage independent, but can couple to an H+ conductance
Publicado: (1995)

Complex Formation between NheB and NheC Is Necessary to Induce Cytotoxic Activity by the Three-Component Bacillus cereus Nhe Enterotoxin
por: Heilkenbrinker, Uta, et al.
Publicado: (2013)

Structure of the NheA Component of the Nhe Toxin from Bacillus cereus: Implications for Function
por: Ganash, Magdah, et al.
Publicado: (2013)

Determining minimal clinically important differences in the North Star Ambulatory Assessment (NSAA) for patients with Duchenne muscular dystrophy
por: Ayyar Gupta, Vandana, et al.
Publicado: (2023)

Upper limb disease evolution in exon 53 skipping eligible patients with Duchenne muscular dystrophy
por: Lilien, Charlotte, et al.
Publicado: (2021)

Role of NHE1 in Nociception
por: Torres-López, Jorge Elías, et al.
Publicado: (2013)

Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy
por: Muntoni, Francesco, et al.
Publicado: (2019)

Phosphorylation of NHE3-S(719) regulates NHE3 activity through the formation of multiple signaling complexes
por: Sarker, Rafiquel, et al.
Publicado: (2017)

Spatial transcriptomics reveal markers of histopathological changes in Duchenne muscular dystrophy mouse models
por: Heezen, L.G.M., et al.
Publicado: (2023)

Rainbow Trout (Oncorhynchus mykiss) Na(+)/H(+) Exchangers tNhe3a and tNhe3b Display Unique Inhibitory Profiles Dissimilar from Mammalian NHE Isoforms
por: Blair, Salvatore, et al.
Publicado: (2021)

Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy
por: Frank, Diane E., et al.
Publicado: (2020)

Glucocorticoids reduce renal NHE8 expression
por: Joseph, Catherine, et al.
Publicado: (2013)

Myocardial Impact of NHE1 Regulation by Sildenafil
por: Escudero, Daiana S., et al.
Publicado: (2021)

Na(+)/H(+) exchanger NHE1 and NHE2 have opposite effects on migration velocity in rat gastric surface cells
por: Paehler vor der Nolte, Anja, et al.
Publicado: (2017)

Downregulating NHE-1 decreases the apoptosis of hippocampal cells in epileptic model rats based on the NHE-1/calpain1 pathway
por: Peng, Shuang, et al.
Publicado: (2023)

Early onset hereditary neuronopathies: an update on non-5q motor neuron diseases
por: Zambon, Alberto A, et al.
Publicado: (2022)

Gene therapy in Duchenne muscular dystrophy: Identifying and preparing for the challenges ahead
por: Heslop, Emma, et al.
Publicado: (2021)

FUNCTIONAL EVALUATION OF AUTISM-ASSOCIATED MUTATIONS IN NHE9
por: Kondapalli, Kalyan C., et al.
Publicado: (2013)

Advances in research on the regulatory mechanism of NHE1 in tumors
por: Hu, Yanxia, et al.
Publicado: (2021)

Analysis of public RNA-sequencing data reveals biological consequences of genetic heterogeneity in cell line populations
por: Fasterius, Erik, et al.
Publicado: (2018)

TCTEX1D1 is a genetic modifier of disease progression in Duchenne muscular dystrophy
por: Spitali, Pietro, et al.
Publicado: (2020)

Development of a novel startle response task in Duchenne muscular dystrophy
por: Maresh, Kate, et al.
Publicado: (2022)

Role of the Na(+)/H(+) exchanger 3 in angiotensin II-induced hypertension in NHE3-deficient mice with transgenic rescue of NHE3 in small intestines
por: Li, Xiao C, et al.
Publicado: (2015)

Pyrazine ring-based Na(+)/H(+) exchanger (NHE) inhibitors potently inhibit cancer cell growth in 3D culture, independent of NHE1
por: Rolver, Michala G., et al.
Publicado: (2020)

Dynamic Na(+)/H(+) exchanger 1 (NHE1) – calmodulin complexes of varying stoichiometry and structure regulate Ca(2+)-dependent NHE1 activation
por: Sjøgaard-Frich, Lise M, et al.
Publicado: (2021)

Multiomic characterization of disease progression in mice lacking dystrophin
por: Signorelli, Mirko, et al.
Publicado: (2023)

A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures
por: Hiller, Monika, et al.
Publicado: (2018)

Growth pattern trajectories in boys with Duchenne muscular dystrophy
por: Stimpson, Georgia, et al.
Publicado: (2022)

Mechanisms of the Regulation of the Intestinal Na(+)/H(+)Exchanger NHE3
por: He, Peijian, et al.
Publicado: (2010)

Cannot write session to /tmp/vufind_sessions/sess_ptsra58thnmjriavt382hftigu