Results 91 to 100 of about 2,067 (154)

PE-068 Impacto econômico nas famílias de crianças com atrofia muscular espinhal tipo 1 no Brasil: um estudo transversal

Jornal de Assistência Farmacêutica e Farmacoeconomia
Introdução: A atrofia muscular espinhal (AME) é uma doença neuromuscular rara, progressiva e debilitante. Apesar dos subsídios para medicamentos, a AME tipo I pode gerar despesas catastróficas para as famílias dos pacientes afetados [1–3]. Objetivo: Este
André Soares Motta-Santos, Kenya Noronha, Carla Barros Reis, Daniela Almeida Freitas, Lélia Maria de Almeida Carvalho, Lívio Matheus Oliveira Silva, Ney Cristian Amaral Boa-Sorte, Mônica Viegas Andrade   +7 more
doaj   +1 more source

Reduction in Perioperative Risk in Patients with Spinal Muscular Atrophy Following the Release of Disease-Modifying Therapies: An Analysis of the National Surgical Quality Improvement Program Database

Children
Background/Objectives: Spinal muscular atrophy (SMA) is a progressive neurodegenerative disease resulting in proximal muscle weakness and paralysis. SMA treatment has radically changed in the past 10 years thanks to the development of novel therapies ...
Erin Toaz, Nisha Pinto, Keith Kilner, Eric Cheon   +3 more
doaj   +1 more source

Clinicogenetic characterization and response to disease-modifying therapies in spinal muscular atrophy: real-world experience from a reference center in Southern Brazil

Jornal de Pediatria
Objective: Spinal Muscular Atrophy linked to chromosome 5q (SMA) is an autosomal recessive neurodegenerative disease characterized by progressive proximal muscle atrophy and weakness.
Ana Letícia Amorim de Albuquerque, Júlia Kersting Chadanowicz, Isabela Possebon Bevilacqua, Ana Lucia Portella Staub, Pablo Brea Winckler, Patricia Zambone da Silva, Simone Chaves Fagondes, Renata Salatti Ferrari, Claudia Denise de Oliveira Trojahn, Viviane Zechlinski Sacharuk, Thayne Woycinck Kowalski, Karina Carvalho Donis, Michele Michelin Becker, Jonas Alex Morales Saute   +13 more
doaj   +1 more source

Clinical decision making around commercial use of gene and genetic therapies for spinal muscular atrophy

Neurotherapeutics
Spinal muscular atrophy is no longer a leading cause of inherited infant death in the United States. Since 2016, three genetic therapies have been approved for the treatment of spinal muscular atrophy.
Megan A. Waldrop
doaj   +1 more source

Post-Marketing Safety of Spinal Muscular Atrophy Therapies: Analysis of Spontaneous Adverse Drug Reactions from EudraVigilance

Background/Objectives: Spinal muscular atrophy (SMA) treatment has evolved with the approval of nusinersen, onasemnogene abeparvovec, and risdiplam. This study aims to assess the post-marketing safety profile of these therapies through the spontaneous ...
Belančić, Andrej, Kovačić Bytyqi, Barbara, Vitezić, Dinko, Mas, Petar, Miletić, Lara, Barbara Kovačić Bytyqi, Petar Mas, Lara Miletić, Dinko Vitezić, Andrej Belančić   +9 more
core   +1 more source

Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan. [PDF]

Ann Clin Transl Neurol
Saito K, Benguerba K, Tsuchida K, Yazawa K, Tsumiyama I, Kayama H, Reyna SP, Khan F, Finkel RS.   +8 more
europepmc   +1 more source

Determinants of hepatic enzyme elevations following onasemnogene abeparvovec: Results from a unified immunomodulation protocol. [PDF]

J Neuromuscul Dis
Mundada V, Parashar S, Dhawan A.
europepmc   +1 more source

Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland - part II - expert based recommendations for surveillance and management of side-effects. [PDF]

J Neuromuscul Dis
Ziegler A, Weiß C, Vill K, Baumann M, Bernert G, Blaschek A, Eisenkölbl A, Flotats-Bastardas M, Friese J, Holzwarth D, Ganter C, Goldhahn K, Hahn A, von der Hagen M, Hartmann H, Hasselmann O, Horber V, Husain RA, Illsinger S, Jacquier D, Johannsen J, Köhler C, Kölbel H, Kolodzig M, Klein A, Pechmann A, von Moers A, Müller-Felber W, Rauscher C, Schreiber G, Schwartz O, Sproß J, Stettner GM, Stoltenburg C, Stumpe E, Trollmann R, Wiegand G, Wilichowski E, Schara U, Kirschner J, collaborators and members of the INTEGRATE ATMP consortium.   +40 more
europepmc   +1 more source

Risdiplam Add-On Therapy Following Onasemnogene Abeparvovec in Children With Spinal Muscular Atrophy and 2 SMN2 Copies: A Multi-Center Case Series. [PDF]

Muscle Nerve
Stoltenburg C, Goldhahn K, von Moers A, Kaindl AM, Weiß C.   +4 more
europepmc   +1 more source

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