Results 131 to 140 of about 2,700 (197)

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

Orphanet Journal of Rare Diseases
Background Feeding and swallowing deficits are reported across the spectrum of spinal muscular atrophy (SMA), with more profound symptoms associated with more severe disease.
Yasmina Martí, Ksenija Gorni, Sandhya Kumari, Anadi Mahajan, Giovanni Baranello, Liesbeth De Waele, Katlyn E. McGrattan   +6 more
doaj   +1 more source

Treatment for Spinal Muscular Atrophy Using Onasemnogene Abeparvovec [PDF]

, 2022
Can Ebru Bekircan‐Kurt, Megan A. Waldrop, Anne M. Connolly, Jerry R. Mendell   +3 more
openalex   +1 more source

Descrição de dados de evolução clínica de pacientes com atrofia muscular espinhal 5q tipo 1 antes e após o uso de Nusinersena e/ou de Onasemnogene abeparvovec [PDF]

Introduction: Spinal muscular atrophy 5q type 1 (SMA 5q type 1) is a neurodegenerative disease characterized by progressive muscle atrophy, hypotonia, and weakness.
Alves, Brenda Klemm Arci Mattos de Freitas
core  

Patient and Caregiver Outcomes After Onasemnogene Abeparvovec Treatment: Findings from the Cure SMA 2021 Membership Survey. [PDF]

Adv Ther, 2023
Toro W, Yang M, Georgieva M, Anderson A, LaMarca N, Patel A, Akbarnejad H, Dabbous O.   +7 more
europepmc   +1 more source

P07 Onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy (SMA): from bench to bedside [PDF]

, 2020
P. Kaufmann, K. Foust, Jonathan Loukes, Jerry R. Mendell   +3 more
openalex   +1 more source

Neurodevelopmental Comorbidities in Early-Onset 5q-SMA Treated with Disease-Modifying Therapies: A Scoping Review [PDF]

Recent studies have reported possible neurodevelopmental comorbidities in some early onset 5q-spinal muscular atrophy (5q-SMA) patients treated with disease-modifying therapies (DMT).
Konop, Carson
core   +1 more source

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

Author Correction: Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial. [PDF]

Nat Med
Kwon JM, Munell F, Le Goff L, Yuge K, Kato T, Cances C, De Waele L, Woodcock IR, Mercuri EM, Proud CM, Darras BT, Hayes LH, Oskoui M, Visootsak J, Williams G, Ilić A, Yang L, van der Pol WL.   +17 more
europepmc   +1 more source

Erratum to: Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment

, 2023
Katlyn McGrattan, Richard D. Shell, Rebecca Hurst-Davis, Sally Dunaway Young, E. A. O’Brien, Arseniy Lavrov, Shiri Wallach, Nicole LaMarca, Sandra P. Reyna, Basil T. Darras   +9 more
openalex   +2 more sources

Onasemnogene Abeparvovec-xioi Gene-Replacement Therapy for Spinal Muscular Atrophy Type 1: Pulmonary and Ventilatory Findings from the Pivotal Phase 3 US Study (STR1VE) [PDF]

, 2020
Richard Shell, John Day, Claudia A. Chiriboga, Thomas O. Crawford, Basil T. Darras, Richard S. Finkel, Anne M. Connolly, Susan Iannaconne, N. Kuntz, Loren D.M. Peña, Perry B. Shieh, Edward C. Smith, J.M. Kwon, Craig M. Zaidman, Megan L. Schultz, Douglas E. Feltner, Sitra Tauscher‐Wisniewski, Francis G. Ogrinc, P. Thomasma, Haojun Ouyang, Thomas A. Macek, Elaine Kernbauer, Douglas M. Sproule, J. Mendell   +23 more
openalex   +1 more source