Results 131 to 140 of about 6,546 (245)

Spinal muscular atrophy and acute lymphoblastic leukemia - is it just a coincidence? [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.
Jovanović Kristina, Dimić Jovana, Brkušanin Miloš, Nikolić Dejan, Škorić Dejan   +4 more
doaj   +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

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

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

Cost-Effectiveness Analysis of Newborn Screening for Spinal Muscular Atrophy in Italy. [PDF]

Background and Objective Untreated spinal muscular atrophy (SMA) is the leading genetic cause of death in children younger than 2 years of age. Early detection through newborn screening allows for presymptomatic diagnosis and treatment of SMA.
Bischof M, Ghetti G, Marcellusi A, Mennini FS, Pane M., Pistillo GM   +5 more
core   +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

Safety analysis of laboratory parameters in paediatric patients with spinal muscular atrophy treated with nusinersen

BMC Pediatrics
Background Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder that can be treated with intrathecal nusinersen, an antisense oligonucleotide.
Xiaomei Zhu, Hui Li, Chaoping Hu, Min Wu, Shuizhen Zhou, Yi Wang, Wenhui Li   +6 more
doaj   +1 more source

An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials [PDF]

, 2019
Basil T. Darras, Michelle A. Farrar, Eugenio Mercuri, Richard S. Finkel, Richard Foster, Steven G. Hughes, Ishir Bhan, Wildon Farwell, Sarah Gheuens   +8 more
openalex   +1 more source

4CPS-131 Effectiveness of nusinersen in paediatric patients SMA1 and SMA2 [PDF]

, 2020
GB Ortenzi, G Palmas, E Andresciani, AMF Garzone, MA Berardi, Sabrina Siliquini, L Porfiri, MS Demeo, S Guglielmi, Carlo Polidori, A Pompilio   +10 more
openalex   +1 more source

Muscle microRNAs in the cerebrospinal fluid predict clinical response to nusinersen therapy in type II and type III spinal muscular atrophy patients [PDF]

, 2021
Iddo Magen, Sharon Aharoni, Nancy‐Sarah Yacovzada, Itay Tokatly Latzer, Christiano R. R. Alves, Liora Sagi, Aviva Fattal‐Valevski, Kathryn J. Swoboda, Jacob Katz, Elchanan Bruckheimer, Yoram Nevo, Eran Hornstein   +11 more
openalex   +1 more source