Results 161 to 170 of about 6,546 (245)

Real-world Adherence to Nusinersen in Adults with Spinal Muscular Atrophy in the US: A Multi-site Chart Review Study

, 2022
Lauren Elman, Bora Youn, Crystal M. Proud, Margaret R. Frey, Senda Ajroud‐Driss, Mary McCormick, David Michelson, Michael S. Cartwright, Terry Heiman‐Patterson, Joseph M. Choi, Aastha Chandak, Artak Khachatryan, Marta Martínez‐Avilés, Angela D. Paradis   +13 more
openalex   +2 more sources

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

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

ND2 COST-UTILITY ANALYSIS OF SINGLE DOSE GENE-REPLACEMENT THERAPY FOR SPINAL MUSCULAR ATROPHY TYPE 1 COMPARED TO CHRONIC NUSINERSEN TREATMENT

, 2019
Daniel C. Malone, Rebecca Dean, Beckley Miller, R. Arjunji, Douglas E. Feltner, D.M. Sproule, I. Jensen, B. Maru, Omar Dabbous   +8 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

, 2022
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   +2 more sources

Nusinersen Use in Spinal Muscular Atrophy [PDF]

Pediatrics, 2019
openaire   +2 more sources

A historical and current review of newborn screening for neuromuscular disorders from around the world: lessons for the United States [PDF]

, 2017
Clarke, Angus John, Ross, Lainie Friedman   +1 more
core   +2 more sources

Respiratory effects of nusinersen treatment in pediatric patients with spinal muscular atrophy types 2 and 3. [PDF]

Eur J Pediatr
Rochman M, Shay Levanon D, Amirav I, Be'er M, Cahal M, Besor O, Fattal-Valevski A, Lavie M.   +7 more
europepmc   +1 more source

Nusinersen versus sham control in later-onset spinal muscular atrophy [PDF]

, 2018
Connolly, A. M., et al,
core   +1 more source

Novel Complication of Nusinersen Treatment: Hyponatremia [PDF]

Journal of Clinical Intensive Care and Medicine, 2018
openaire   +1 more source

CO108 Longitudinal Trends in Hospitalization Rates in Patients with Later-Onset Spinal Muscular Atrophy (SMA) Treated with Nusinersen up to 4.5 Years: Results from the Cherish and Shine Studies

, 2023
Tao Jiang, Basil T. Darras, Bora Youn, C. Makepeace, R. Chin, Z. Berger, Stephanie Raynaud, Angela D. Paradis, Nicole B. Johnson   +8 more
openalex   +1 more source