Results 181 to 190 of about 7,776 (285)

Exploring the Influence of Concurrent Nutritional Therapy on Children with Spinal Muscular Atrophy Receiving Nusinersen Treatment

Children
Background This study examines spinal muscular atrophy (SMA), a neuromuscular disease associated with malnutrition. Our goals are to assess how effectively screening tools can detect malnutrition and evaluate the impact of nutritional interventions on ...
Eymen Pinar, Bilal Berke Ayvaz, Erkan Akkus, Ipek Ulkersoy, T. Dilek, Yılmaz Zindar, Fitnat Uluğ, Aysel Guzeler, Hüseyin Kılıç, Serhat Guler, O. F. Beser, S. Saltık, F. Cullu Cokugras   +12 more
semanticscholar   +1 more source

Compilation of online resources of relevance to 'Spinraza and advanced therapies:a stakeholder special' issue of Gene Therapy [PDF]

, 2017
Prakash, V, Yáñez-Muñoz, R J
core   +1 more source

Onasemnogene Abeparvovec Treatment after Nusinersen in an Infant with Spinal Muscular Atrophy Type 1.

The Kurume Medical Journal
BACKGROUND Until recently, the treatment of spinal muscular atrophy (SMA) was limited to symptomatic treatment with no cure. Three innovative drugs, nusinersen, onasemnogene abeparvovec (OA), and risdiplam have been developed to treat SMA.
Daiki Nanri, K. Yuge, Kohei Goto, Takuro Kimura, Yukako Yae, T. Mizuochi, Ryosuke Sato, Tomoyo Itonaga, T. Maeda, Yushiro Yamashita   +9 more
semanticscholar   +1 more source

Respiratory outcomes post nusinersen in spinal muscular atrophy type 1

, 2020
Kate Gonski, Dominic A. Fitzgerald
openalex   +1 more source

Intrathecal administration of Nusinersen in children and adolescent SMA type 1 and 2 [PDF]

, 2023
Michele Michelin Becker, Lygia Ohlweiler, Josiane Ranzan, Hugo Leonardo Justo Horácio, Ana Clara Bernardi Saul, Layanna Bezerra Maciel Pereira, Renata Yasmin Cardoso Sousa, Dayana de Lima Mariano, Rudimar dos Santos Riesgo   +8 more
openalex   +1 more source

Why antisense could make sense for neurodegeneration [PDF]

, 2017
Massey, Thomas, Robertson, Neil
core   +1 more source

Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study

, 2019
Darryl C. De Vivo, Enrico Bertini, Kathryn J. Swoboda, Wuh‐Liang Hwu, Thomas O. Crawford, Richard S. Finkel, Janbernd Kirschner, Nancy L. Kuntz, Julie Parsons, Monique M. Ryan, Russell J. Butterfield, Haluk Topaloğlu, Tawfeg Ben‐Omran, Valeria Sansone, Yuh‐Jyh Jong, Francy Shu, John F. Staropoli, Douglas A. Kerr, Alfred Sandrock, Christopher Stebbins, Marco Petrillo, Gabriel Braley, Kristina Johnson, Richard Foster, Sarah Gheuens, Ishir Bhan, Sandra P. Reyna, Stephanie Fradette, Wildon Farwell   +28 more
openalex   +1 more source

Clinical Outcome of Adult Spinal Muscular Atrophy Patients Treated with Nusinersen: A Case Series Review

, 2020
Keng Lam, Abel Wu
openalex   +2 more sources

Protein production is an early biomarker for RNA‐targeted therapies [PDF]

, 2018
Alex, Jacob, Barthélemy, Nicolas, Bateman, Randall J, Bollinger, James G, Cole, Tracy, Kordasiewicz, Holly B, Miller, Timothy M, Sato, Chihiro, Schoch, Kathleen M, Self, Wade K, Swayze, Eric   +10 more
core   +2 more sources

Gene Therapy, more than ever-a new vision for the journal [PDF]

, 2017
Yáñez-Muñoz, R J
core   +1 more source