Results 141 to 150 of about 6,859 (228)

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

Maria Liguori, Annalisa Bianco, Alessandro Introna, Arianna Consiglio, Giammarco Milella, Elena Abbatangelo, Eustachio D’Errico, Flavio Licciulli, Giorgio Grillo, Isabella Laura Simone   +9 more
openalex   +1 more source

Swallowing and feeding after disease-modifying treatment for spinal muscular atrophy: a systematic review of assessment modalities and outcomes. [PDF]

Orphanet J Rare Dis
Martí Y, Gorni K, Kumari S, Mahajan A, Baranello G, Waele L, McGrattan KE.   +6 more
europepmc   +1 more source

Assessment of safety and efficacy of risdiplam treatment in adults with spinal muscular atrophy. [PDF]

Front Neurol
Jaworek A, Jira K, Allen M, Zhao S, Kelly K, Moravec T, Tellez M, Heintzman S, Reynolds J, Sterling G, Kolb SJ, Arnold WD, Elsheikh B.   +12 more
europepmc   +1 more source

Nusinersen for spinal muscular atrophy [PDF]

Therapeutic Advances in Neurological Disorders, 2018
Claudia D. Wurster, Albert C. Ludolph
openaire   +3 more sources

Pharmacokinetics of therapies approved for spinal muscular atrophy: A narrative review of current evidence. [PDF]

J Int Med Res
Sel EK, Gkrinia EMM, Roncato R, Vitezić D, Janković S, Belančić A, EACPT Early Career Clinical Pharmacologists Working Group.   +6 more
europepmc   +1 more source

Nusinersen treatment in adults with severe spinal muscular atrophy: A real-life retrospective observational cohort study

, 2022
Claire Lefeuvre, M. Brisset, M. Sarlon, Núria Mestres Petit, David Orlikowski, Bernard Clair, T. Thiry, RY Carlier, Hélène Prigent, Gaël Nicolas, Djillali Annane, Pascal Laforêt, Samuel Pouplin   +12 more
openalex   +2 more sources

Cost-Effectiveness of Onasemnogene Abeparvovec Compared With Nusinersen and Risdiplam in Patients With Spinal Muscular Atrophy Type 1 in Brazil [PDF]

Brígida Dias Fernandes, Fernanda d’Athayde Rodrigues, Hérica Núbia Cardoso Cirilo, Stéfani Sousa Borges, Bárbara Krug, Lívia Fernandes Probst, Ivan Ricardo Zimmermann   +6 more
openalex   +1 more source

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: A Novel Antisense Oligonucleotide for the Treatment of Spinal Muscular Atrophy

, 2019
Erin E. Neil, Elizabeth K. Bisaccia
openalex   +2 more sources

Diverging Safety Signals: A Trend Analysis of Suspected Adverse Drug Reactions Reporting for Spinal Muscular Atrophy Therapies in the European Union. [PDF]

Neurol Int
Belančić A, Mas P, Stević I, Vitezić D, Janković S.   +4 more
europepmc   +1 more source