Zobrazeno 1 - 10 of 42 pro vyhledávání: '"Sindhu Ramchandren"'
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Akademický článek
Generation of Duchenne muscular dystrophy patient-specific induced pluripotent stem cell line lacking exons 45–50 of the dystrophin gene (IITi001-A)
Autor: Binyamin Eisen, Ronen Ben Jehuda, Ashley J. Cuttitta, Lucy N. Mekies, Irina Reiter, Sindhu Ramchandren, Michael Arad, Daniel E. Michele, Ofer Binah
Publikováno v: Stem Cell Research, Vol 29, Iss , Pp 111-114 (2018)
Duchenne muscular dystrophy (DMD) is an X-linked progressive muscle degenerative disease caused by mutations in the dystrophin gene. We generated induced pluripotent stem cells (iPSCs) from a 13-year-old male patient carrying a deletion mutation of e
Externí odkaz: https://doaj.org/article/d79c103b1f9f407b90f27d64178fe354
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5
Neuropathy due to bi-allelic SH3TC2 variants: genotype-phenotype correlation and natural history
Autor: Tyler Rehbein, Tong Tong Wu, Simona Treidler, Davide Pareyson, Richard Lewis, Sabrina W Yum, Brett A McCray, Sindhu Ramchandren, Joshua Burns, Jun Li, Richard S Finkel, Steven S Scherer, Stephan Zuchner, Michael E Shy, Mary M Reilly, David N Herrmann
Publikováno v: Brain.
Recessive SH3TC2 variants cause Charcot-Marie-Tooth disease type 4C (CMT4C). CMT4C is typically a sensorimotor demyelinating polyneuropathy, marked by early onset spinal deformities, but its clinical characteristics and severity are quite variable. C
Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::a26dd562c85ee68a6daeb6da4caa1e03
https://doi.org/10.1093/brain/awad095
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7
Vibrance-mg: Clinical Trial of Nipocalimab in Pediatric Myasthenia Gravis
Autor: Sindhu Ramchandren, Shawn Black, Hong Sun
Publikováno v: Neurology. 99:S41.1-S41
ObjectiveWe describe an open-label study of nipocalimab to determine the effect of nipocalimab in pediatric participants with gMG.BackgroundNipocalimab is a high affinity, fully human, aglycosylated, effectorless IgG1 anti FcRn monoclonal antibody th
Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::d7d7fd084679415d3b0f4db9753e6ac0
https://doi.org/10.1212/01.wnl.0000903324.87754.45
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8
Vivacity MG Phase 3 Study: Clinical Trial of Nipocalimab Administered to Adults With Generalized Myasthenia Gravis
Autor: Sindhu Ramchandren, Panna Sanga, Michel Burcklen, Hong Sun
Publikováno v: Neurology. 99:S41.2-S41
ObjectiveWe describe Vivacity-MG3. our pivotal Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Nipocalimab Administered to Adults With gMG
Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::706a4ce0b9d0fa60d70ad03417a5382f
https://doi.org/10.1212/01.wnl.0000903328.46907.49
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9
Development and Validation of the Pediatric Charcot-Marie-Tooth Disease Quality of Life Outcome Measure
Autor: Shawna M. E. Feely, Mary M. Reilly, Michael E. Shy, Tong Tong Wu, Richard S. Finkel, Carly E. Siskind, Joshua Burns, T Estilow, Sindhu Ramchandren
Publikováno v: Annals of neurologyReferences. 89(2)
OBJECTIVE Charcot-Marie-Tooth disease (CMT) reduces health-related quality of life (QOL), especially in children. Defining QOL in pediatric CMT can help physicians monitor disease burden clinically and in trials. We identified items pertaining to QOL
Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e5614c77c199f3a03ac30a2782c68cb6
https://pubmed.ncbi.nlm.nih.gov/33222249
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