Results 231 to 240 of about 139,920 (262)

Onasemnogene Abeparvovec in Type I Spinal Muscular Atrophy: 24‐Month Follow‐Up From the Italian Registry

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Onasemnogene abeparvovec (OA) is an AAV9‐based gene therapy for spinal muscular atrophy type I (SMA I). Real‐world outcomes show increased response variability compared to clinical trials, and follow‐up data beyond 12–18 months are limited.
Marika Pane, Giorgia Coratti, Chiara Cutrì, Antonio Varone, Riccardo Masson, Adele D'Amico, Valeria Sansone, Sonia Messina, Federica Ricci, Chiara Ticci, Claudio Bruno, Caterina Agosto, Francesca Benedetti, Antonella Pini, Sabrina Siliquini, Massimiliano Filosto, Alberto Zambon, Ilaria Bitetti, Maria Rosaria Manna, Claudia Dosi, Riccardo Zanin, Stefano Parravicini, Roberto De Sanctis, Giulia Stanca, Michela Catteruccia, Michele Tosi, Irene Mizzoni, Emilio Albamonte, Valentina Franchino, Maria Sframeli, Ilaria Cavallina, Elena Procopio, Michele Sacchini, Simone Morando, Noemi Brolatti, Federica Trucco, Gaia Scarpini, Elena Briganti, Beatrice Berti, Concetta Palermo, Daniela Leone, Stefano C. Previtali, Eugenio Mercuri, the ITASMAC working group   +43 more
wiley   +1 more source

Numerical study on the impact of coal fractures on seismic wave dispersion and attenuation: anisotropic WIFF effects. [PDF]

Sci Rep
Li B, Zou G, Wang J, Chen H, Zhu G.
europepmc   +1 more source

Predictive wave engineering in polymer phononic materials <i>via</i> viscoelastic-geometric coupling.

Mater Horiz
Beniwal S, Bose RK, Krushynska AO.
europepmc   +1 more source

Novel photonic crystal fibre for low-noise coherent supercontinuum generation. [PDF]

Sci Rep
Morel R, Millo J, Forget N, Thibaut V, Marcadier M, Jullien A, Pertot Y, Cassez A, Andrieux V, Labat D, Vanvincq O, Kudlinski A, Dudley JM, Sylvestre T.   +13 more
europepmc   +1 more source

Hybrid PP composites reinforced with banana midrib fiber and rGO for enhanced mechanical and dielectric performance. [PDF]

Sci Rep
Al-Amin M, Ullah MH, Rahman MM, Alam MK, Khan NI, Rahman MJ.   +5 more
europepmc   +1 more source

Harnessing normal-shear coupling in metabarriers for deep sub-wavelength underwater noise control. [PDF]

NPJ Acoust
Dal Poggetto VF, Miniaci M.
europepmc   +1 more source

Experimental study on electrical dispersion in 3D printed resin cores reveals pore and fluid property mechanisms. [PDF]

Sci Rep
Shi H, Ke S, Zhang Y, Hu H, Luo H.
europepmc   +1 more source

Copolymer Considerations on the Displacing Effect for the Effective Functionalization of 3D-Printed Photopolymer Sensors with Carbon Nanotubes. [PDF]

ACS Omega
Sevriugina V, Lepcio P.
europepmc   +1 more source