Results 61 to 70 of about 3,107 (205)

Safety and tolerability of onasemnogene abeparvovec for patients with spinal muscular atrophy weighing ≤17 kg and ≤24 months old from OFELIA, a phase 4, open-label, multicenter, non-randomised, interventional study. [PDF]

Lancet Reg Health Am
Saute JA, Muntadas J, Gurgel-Giannetti J, Monges S, Aliberti P, Mendonça RH, Alecu I, Ritter S, Martins de Lana J, Mumneh N, Zanoteli E, OFELIA Study Group.   +11 more
europepmc   +2 more sources

Systematic literature review of the economic burden of spinal muscular atrophy and economic evaluations of treatments

Orphanet Journal of Rare Diseases, 2021
Background Spinal muscular atrophy (SMA) is a rare and devastating condition for which new disease-modifying treatments have recently been approved. Given the increasing importance of economic considerations in healthcare decision-making, this review ...
Tamara Dangouloff, Camille Botty, Charlotte Beaudart, Laurent Servais, Mickaël Hiligsmann   +4 more
doaj   +1 more source

Gene therapy-based strategies for spinal muscular atrophy—an Asia-Pacific perspective

Molecular and Cellular Pediatrics, 2023
Onasemnogene abeparvovec has been life-changing for children with spinal muscular atrophy (SMA), signifying the potential and progress occurring in gene- and cell-based therapies for rare genetic diseases.
Michelle A. Farrar, Loudella Calotes-Castillo, Ranil De Silva, Peter Barclay, Lani Attwood, Julie Cini, Monica Ferrie, Didu S. Kariyawasam   +7 more
doaj   +1 more source

Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial

Nature Medicine, 2022
SPR1NT (NCT03505099) was a Phase III, multicenter, single-arm study to investigate the efficacy and safety of onasemnogene abeparvovec for presymptomatic children with biallelic SMN1 mutations treated at ≤6 weeks of life.
K. Strauss, M. Farrar, F. Muntoni, K. Saito, J. Mendell, L. Servais, H. McMillan, R. Finkel, K. Swoboda, J. Kwon, C. Zaidman, C. Chiriboga, S. Iannaccone, J. Krueger, J. Parsons, P. Shieh, S. Kavanagh, S. Tauscher‐Wisniewski, B. McGill, T. Macek   +19 more
semanticscholar   +1 more source

Bridging the Gap : Gene Therapy in a Spinal Muscular Atrophy Type 1 Patient [PDF]

, 2022
Molecular therapies exploit understanding of pathogenic mechanisms to reconstitute impaired gene function or manipulate flawed RNA expression. These therapies include 1) RNA interference by antisense oligonucleotides, 2) mRNA modification using small ...
Corti, Stefania, Costamagna, Gianluca, Govoni, Alessandra, Wise, Adina   +3 more
core   +1 more source

Onasemnogene Abeparvovec Treatment after Nusinersen in an Infant with Spinal Muscular Atrophy Type 1

The Kurume Medical Journal, 2022
BACKGROUND Until recently, the treatment of spinal muscular atrophy (SMA) was limited to symptomatic treatment with no cure. Three innovative drugs, nusinersen, onasemnogene abeparvovec (OA), and risdiplam have been developed to treat SMA.
DAIKI NANRI, Kotaro Yuge, KOHEI GOTO, Takuro Kimura, Yukako Yae, Tatsuki Mizuochi, Ryosuke Sato, Tomoyo Itonaga, Tomoki Maeda, Yushiro Yamashita   +9 more
openalex   +3 more sources

Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma^®) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System. [PDF]

Pharmaceuticals (Basel)
The recent introduction of the innovative therapy, onasemnogene abeparvovec (Zolgensma®), has revolutionized the spinal muscular atrophy (SMA) therapeutic landscape.
Ruggiero R, Balzano N, Nicoletti MM, di Mauro G, Fraenza F, Campitiello MR, Rossi F, Capuano A.   +7 more
europepmc   +2 more sources

Case report: A case of spinal muscular atrophy in a preterm infant: risks and benefits of treatment

Frontiers in Neurology, 2023
Spinal muscular atrophy (SMA) is a neuromuscular genetic disorder caused by the loss of lower motor neurons leading to progressive muscle weakness and atrophy.
Elisa Nigro, Eyal Grunebaum, Binita Kamath, Christoph Licht, Caroline Malcolmson, Aamir Jeewa, Craig Campbell, Hugh McMillan, Pranesh Chakraborty, Mark Tarnopolsky, Hernan Gonorazky   +10 more
doaj   +1 more source

Cost-effectiveness of treatments for presymptomatic newborn patients with spinal muscular atrophy and two or three copies of the survival motor neuron 2 gene in Italy [PDF]

ObjectiveWe assessed the cost effectiveness of onasemnogene abeparvovec (OA) for presymptomatic infants with two or three copies of the survival motor neuron 2 (SMN2) gene (diagnosed/treated <= 6 weeks old) who lack functional SMN1 gene (biallelic ...
Basile, M, Di Brino, E, Ghetti, G, Pane, M, Rumi, F, Valentini, I   +5 more
core   +1 more source

Thrombotic microangiopathy following onasemnogene abeparvovec for spinal muscular atrophy: A case series [PDF]

, 2021
Spinal muscular atrophy is treated with onasemnogene abeparvovec, which replaces the missing survival motor neuron 1 gene via an adeno-associated virus vector.
Arya, Kapil, Chand, Deepa H, Dandamudi, Raja, Dharnidharka, Vikas R, Farrar, Michelle A, Goedeker, Natalie L, Mackie, Fiona E, Millner, Rachel, Reyna, Sandra P, Zaidman, Craig   +9 more
core   +1 more source