Results 41 to 50 of about 3,107 (205)

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

Nature Medicine
Jennifer M. Kwon, Francina Munell, Laure Le Goff, Kotaro Yuge, Tamaki Kato, Claude Cancés, Liesbeth De Waele, Ian Woodcock, Eugenio Mercuri, Crystal M. Proud, Basil T. Darras, Leslie H. Hayes, Maryam Oskoui, Jeannie Visootsak, Gemma Williams, Aleksandra Ilić, Lina Yang, W. Ludo van der Pol   +17 more
openalex   +2 more sources

Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment. [PDF]

J Neuromuscul Dis, 2023
McGrattan KE, Shell RD, Hurst-Davis R, Young SD, O'Brien E, Lavrov A, Wallach S, LaMarca N, Reyna SP, Darras BT.   +9 more
europepmc   +3 more sources

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

J Neuromuscul Dis, 2023
McGrattan KE, Shell RD, Hurst-Davis R, Young SD, O'Brien E, Lavrov A, Wallach S, LaMarca N, Reyna SP, Darras BT.   +9 more
europepmc   +3 more sources

Implications of circulating neurofilamentsfor spinal muscular atrophytreatment early in life: A case series

Molecular Therapy: Methods & Clinical Development, 2021
This longitudinal cohort study aimed to determine whether circulating neurofilaments (NFs) can monitor response to molecular therapies in newborns with spinal muscular atrophy (SMA; NCT02831296).
Christiano R.R. Alves, Marco Petrillo, Rebecca Spellman, Reid Garner, Ren Zhang, Michael Kiefer, Sarah Simeone, Jihee Sohn, Eric J. Eichelberger, Emma Rodrigues, Elizabeth A. Arruda, Elise L. Townsend, Wildon Farwell, Kathryn J. Swoboda   +13 more
doaj   +1 more source

Open-label phase IV trial evaluating nusinersen after onasemnogene abeparvovec in children with spinal muscular atrophy. [PDF]

J Clin Invest
BACKGROUND Spinal muscular atrophy (SMA) is a rare genetic neuromuscular disease caused by deletions or mutations of the survival motor neuron 1 (SMN1) gene.
Proud CM, Finkel RS, Parsons JA, Masson R, Brandsema JF, Kuntz NL, Foster R, Li W, Littauer R, Sohn J, Fradette S, Youn B, Paradis AD, RESPOND Study Group.   +13 more
europepmc   +2 more sources

An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER)

Journal of Market Access & Health Policy, 2021
Background: Recent cost-utility analysis (CUA) models for onasemnogene abeparvovec (Zolgensma®, formerly AVXS-101) in spinal muscular atrophy type 1 (SMA1) differ on key assumptions and results.
Rebecca Dean, Ivar Jensen, Phil Cyr, Beckley Miller, Benit Maru, Douglas M. Sproule, Douglas E. Feltner, Thomas Wiesner, Daniel C. Malone, Matthias Bischof, Walter Toro, Omar Dabbous   +11 more
doaj   +1 more source

Administration of Onasemnogene Abeparvovec in an Infant With Spinal Muscular Atrophy and PCR-Confirmed SARS-CoV-2 Infection. [PDF]

Cureus
Onasemnogene abeparvovec (OA) is used to treat spinal muscular atrophy (SMA), and early treatment is critical. However, OA administration may trigger an immune response.
Shimomura I, Maruyama S, Kodama Y, Matsunaga M, Okamoto Y.   +4 more
europepmc   +2 more sources

Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy [PDF]

, 2021
BACKGROUND & AIMS: Spinal muscular atrophy (SMA) is an autosomal recessive, childhood-onset motor neuron disease. Onasemnogene abeparvovec (OA) is a gene therapy designed to address SMA\u27s root cause.
Chand, Deepa, Kaufmann, Petra, Kleyn, Aaron, Kullak-Ublick, Gerd, McMillan, Hugh, Mohr, Franziska, Montgomery, Kyle, Sun, Rui, Tauscher-Wisniewski, Sitra, Tukov, Francis Fonyuy   +9 more
core   +2 more sources

Evaluation of cardiac function in patients with SMA after treatment with onasemnogene abeparvovec [PDF]

Pediatrics International
Spinal muscular atrophy (SMA) is a recessively inherited neurological disease resulting in motor neuron disorder. Onasemnogene abeparvovec is a gene replacement therapy used to treat patients with SMA.
Akihisa Horigome, Satoshi Takasago, Reiko Arakawa, Katsuyoshi Shimozawa, Masao Kaneshige, Keiji Goishi, Hideko Uryu, Junko Yamanaka, Hiroyuki Shichino, Ayumi Mizukami   +9 more
openalex   +2 more sources

Recombinant Adeno-Associated Virus Serotype 9 Gene Therapy in Spinal Muscular Atrophy

Frontiers in Neurology, 2021
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease caused by deletion or mutation of the SMN1 gene. It is characterized by a progressive loss of motor neurons resulting in muscle weakness.
Katarzyna Kotulska, Aviva Fattal-Valevski, Jana Haberlova   +2 more
doaj   +1 more source