Results 161 to 170 of about 7,776 (285)

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

Patients’ Perceptions of Nusinersen Effects according to Their Responder Status

Journal of Clinical Medicine
Background and Objective: Patients with spinal muscular atrophy (SMA) treated with a dis-ease-modifying therapy (DMT) are often classified as responders or non-responders based on at-tainment of a specific improvement threshold on validated functional scales. This categorization may significantly impact treatment reimbursement in some countries.
Lilien, C, Vrscaj, E, Thapaliya, G, Deconinck, N, De Waele, L, Duong, T, Haberlová, J, Kumhera, M, Peirens, G, Szabo, L, Tahon, V, Tang, WJ, Benmhammed, N, Médard, L, Servais, L   +14 more
openaire   +3 more sources

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

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

, 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

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

Correction to: Nusinersen: A Review in 5q Spinal Muscular Atrophy [PDF]

, 2021
Sheridan M. Hoy
openalex   +1 more source

Type 1 spinal muscular atrophy treated with nusinersen in Norway, a five-year follow-up [PDF]

Background: New treatments for 5q spinal muscular atrophy (SMA) have led to changes in the disease phenotype. Questions about long-term efficacy, however, persist.
Hagen, Milada, Holtebekk, Elisabeth, Ramm-Pettersen, Anette, Rasmussen, Magnhild, Wallace, Sean Ciaran, Wik-Klokk, Merete, Zetterberg, Henrik, Ørstavik, Kristin   +7 more
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 [PDF]

, 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

Stabilization of Respiratory Function in Pediatric Spinal Muscular Atrophy Treated with Nusinersen [PDF]

, 2020
Archana Chacko, S. Deegan, Peter D. Sly, Leanne Gauld   +3 more
openalex   +1 more source

Mid- and long-term (at least 12 months) follow-up of patients with spinal muscular atrophy (SMA) treated with nusinersen, onasemnogene abeparvovec, risdiplam or combination therapies: A systematic review of real-world study data [PDF]

, 2022
Judit Erdös, Claudia Wild
openalex   +1 more source