Results 151 to 160 of about 6,546 (245)

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, Anton S. Saul, Layanna Bezerra Maciel Pereira, Renata Yasmin Cardoso Sousa, Dayana de Lima Mariano, Rudimar dos Santos Riesgo   +8 more
openalex   +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

PND9 EVENT-FREE SURVIVAL AND MOTOR MILESTONE ACHIEVEMENT FOLLOWING AVXS-101 AND NUSINERSEN INTERVENTIONS CONTRASTED TO NATURAL HISTORY FOR TYPE I SPINAL MUSCULAR ATROPHY PATIENTS

, 2019
Omar Dabbous, Douglas M. Sproule, Douglas E. Feltner, Francis G. Ogrinc, M. Menier, M. Droege, B. Maru, F. Khan, R. Arjunji   +8 more
openalex   +1 more source

Why antisense could make sense for neurodegeneration [PDF]

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

The role of experiential knowledge within attitudes towards genetic carrier screening : a comparison of people with and without experience of spinal muscular atrophy [PDF]

, 2018
Purpose: Autosomal recessive conditions, whilst individually rare, are a significant health burden with limited treatment options. Population carrier screening has been suggested as a means of tackling them.
Boardman, Felicity K., Griffiths, Frances, Warren, O., Young, P.   +3 more
core  

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

O USO DO NUSINERSEN NO TRATAMENTO DA ATROFIA MUSCULAR ESPINHAL: REVISÃO DE LITERATURA

, 2020
Elzanice Monteiro DOMINGOS, Annalu Moreira Aguiar   +1 more
openalex   +2 more sources