Results 231 to 240 of about 27,936,721 (303)

Toward an effective translational science engine

Clinical and Translational Science
Octavian C. Ioachimescu, Reza Shaker
doaj   +1 more source

Validation of a Cellular Imaging‐Based Method as a Potential Biomarker for SPG4 Hereditary Spastic Paraplegia

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Background Hereditary Spastic Paraplegia (HSP) comprises a group of rare genetic diseases characterized by length‐dependent axonal degeneration of the corticospinal tracts and dorsal columns, whose main clinical feature is spastic gait. Pathogenic variants in the SPG4 gene cause Spastic Paraplegia Type 4 (SPG4‐HSP), the most common form of HSP.
Gaia Fattorini, Valerio Licursi, Gianmarco Dalla Zanna, Flavio Dal Canto, Melissa Barghigiani, Nunzio Setola, Salvatore Rossi, Antonio Funcis, Filippo M. Santorelli, Gabriella Silvestri, Carlo Casali, Francesca Sardina, Cinzia Rinaldo   +12 more
wiley   +1 more source

Re‐Purposing Sapropterin (Kuvan) for ACTA2‐Related Multisystemic Smooth Muscle Dysfunction Syndrome: A Translational Mechanistic and First‐In‐Human Therapeutic Report

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Multisystemic smooth muscle dysfunction syndrome (MSMDS) is an ultra‐rare, ACTA2‐related disorder characterized by severe cerebrovascular disease, aortic aneurysms, and smooth muscle dysfunction. Using molecular dynamics simulations and in silico drug screening, we identified that sapropterin dihydrochloride (Kuvan) is a candidate capable of ...
Moran Hausman‐Kedem, Noa Bielopolski, Vijai Krishnan, Shane Wald‐Altman, Shelly I Shrian, Omer Bar‐Yosef, Livia Kapusta, Claire Shamber, Patricia L. Musolino, Miguel Weil   +9 more
wiley   +1 more source

Mirvetuximab Soravtansine: Mechanism of Action, Clinical and Translational Science. [PDF]

Clin Transl Sci
Menon R, Bako E, Liu S, Stec J, Thakur S, Deutschman E, Ab O, Jain S.   +7 more
europepmc   +1 more source

Establishing a standardized biorepository across 3 clinical and translational science center-affiliated veterinary institutions is feasible and productive. [PDF]

Am J Vet Res
Crawford LK, Church ME, Boudreaux KA, Lapin JN, Hahn LA, Solis LJ, Wilson R, Zetterberg H, Catacci Guimaraes F, Shen S, Toedebusch RG, Vernau K, Dupanloup A, Panek W, Cameron S, Toedebusch CM.   +15 more
europepmc   +1 more source

What's Hot and What's New in Xenotransplantation From the Young Investigator Committee of the IXA-Basic and Translational Science. [PDF]

Xenotransplantation
Goerlich CE, Salvaris EJ, Fischer K, Citro A, Giarraputo A, Ladowski J, Mazancourt ES, Wang L, Eisenson D, Connolly MR, Stern J, Longchamp A, Meier RPH.   +12 more
europepmc   +1 more source