Results 151 to 160 of about 7,776 (285)

Preliminary Safety and Tolerability of a Novel Subcutaneous Intrathecal Catheter System for Repeated Outpatient Dosing of Nusinersen to Children and Adults With Spinal Muscular Atrophy [PDF]

, 2018
Kevin A. Strauss, Vincent J. Carson, Karlla W. Brigatti, Millie Young, Donna L. Robinson, Christine Hendrickson, Michael D. Fox, Robert M. Reed, Erik G. Puffenberger, William G. Mackenzie, Freeman Miller   +10 more
openalex   +1 more source

Administration of nusinersen via paramedian approach for spinal muscular atrophy [PDF]

, 2020
Hideyuki Iwayama, Norimitsu Wakao, Hirokazu Kurahashi, Norika Kubota, Ayako Hattori, Toshiyuki Kumagai, Akihisa Okumura   +6 more
openalex   +1 more source

Nusinersen Improves Motor Function in Type 2 and 3 Spinal Muscular Atrophy Patients across Time

Biomedicines
Spinal muscular atrophy (SMA) is a genetic disorder primarily caused by mutations in the SMN1 gene, leading to motor neuron degeneration and muscle atrophy, affecting multiple organ systems. Nusinersen treatment targets gene expression and is expected to
Bogdana Cavaloiu, Iulia-Elena Simina, Crisanda Vîlciu, Iuliana-Anamaria Trăilă, M. Puiu   +4 more
semanticscholar   +1 more source

Effective local treatment of necrotizing fasciitis using a chlorine solution obtained by electrolysis [PDF]

Srpski Arhiv za Celokupno Lekarstvo
Introduction. Spinal muscular atrophy (SMA) and acute lymphoblastic leukemia (ALL) are rare diseases, with usual onset in childhood. To date, no cases have been reported where these conditions co-exist in one patient.
Kovačević Predrag T.
doaj   +1 more source

Nusinersen treatment of spinal muscular atrophy: current knowledge and existing gaps [PDF]

, 2018
Teresa Gidaro, Laurent Servais
openalex   +1 more source

Serum creatine kinase and creatinine in adult spinal muscular atrophy under nusinersen treatment [PDF]

, 2021
Maren Freigang, Claudia D. Wurster, Tim Hagenacker, Benjamin Stolte, Markus Weiler, Christoph Kamm, Olivia Schreiber‐Katz, Alma Osmanovic, Susanne Petri, Alexander B. Kowski, Thomas Meyer, Jan Christoph Koch, Isabell Cordts, Marcus Deschauer, Paul Lingor, Elisa Aust, Daniel Petzold, Albert C. Ludolph, Björn Falkenburger, Andreas Hermann, René Günther   +20 more
openalex   +1 more source

An early Transcriptomic Investigation in Adult Patients with Spinal Muscular Atrophy Under Treatment with Nusinersen

Journal of Molecular Neuroscience
Spinal muscular atrophy (SMA) is a rare degenerative disorder with loss of motor neurons caused by mutations in the SMN1 gene. Nusinersen, an antisense oligonucleotide, was approved for SMA treatment to compensate the deficit of the encoded protein SMN ...
Maria Liguori, Annalisa Bianco, Alessandro Introna, Arianna Consiglio, Giammarco Milella, Elena Abbatangelo, E. D’Errico, F. Licciulli, Giorgio Grillo, I. Simone   +9 more
semanticscholar   +1 more source

B.06 Safety and efficacy of nusinersen in infants/children with spinal muscular atrophy (SMA): part 1 of the phase 2 EMBRACE study [PDF]

, 2018
PB Shieh, Gyula Acsádi, Wolfgang Mueller‐Felber, T. B. B. Crawford, Randal Richardson, Niranjana Natarajan, Diana Castro, Sarah Gheuens, Ishir Bhan, Giulia Gambino, Peng Sun, Wildon Farwell, S. Reyna, Jiri Vajsar   +13 more
openalex   +1 more source

Nusinersen in type 0 spinal muscular atrophy: should we treat? [PDF]

, 2020
Eloisa Tiberi, Simonetta Costa, Marika Pane, Francesca Priolo, Roberto De Sanctis, Domenico M. Romeo, Francesco Danilo Tiziano, Giorgio Conti, Giovanni Vento, Eugenio Mercuri   +9 more
openalex   +1 more source

Intrathecal Administration of Nusinersen Using the Ommaya Reservoir in an Adult with 5q-Related Spinal Muscular Atrophy Type 1 and Severe Spinal Deformity [PDF]

, 2021
Vasileios Papaliagkas, Nikolaos Foroglou, Petros Toulios, Maria Moschou, Maria Gavriilaki, Konstantinos Notas, Evangelia Chatzikyriakou, Georgia Zafeiridou, Marianthi Arnaoutoglou, Vasilios Κ. Kimiskidis   +9 more
openalex   +1 more source