Results 41 to 50 of about 2,700 (197)

Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy [PDF]

JAMA Neurology, 2021
Jerry R. Mendell, Samiah Al-Zaidy, Kelly J. Lehman, Markus McColly, Linda Lowes, Lindsay N. Alfano, Natalie F. Reash, M. Iammarino, Kathleen R. Church, A. de Kleyn, Matthew N. Meriggioli, Richard Shell   +11 more
openalex   +3 more sources

Eculizumab for Thrombotic Microangiopathy Induced by Onasemnogene Abeparvovec in Spinal Muscular Atrophy. [PDF]

Case Rep Nephrol Dial
Levart TK, Kar NO, Pegan CM, Vrščaj E, Gergeli AT, Loboda T, Osredkar D.   +6 more
europepmc   +3 more sources

Epithelioid neoplasm of the spinal cord in a child with spinal muscular atrophy treated with onasemnogene abeparvovec. [PDF]

Mol Ther, 2023
Retson L, Tiwari N, Vaughn J, Bernes S, Adelson PD, Mansfield K, Libertini S, Kuzmiski B, Alecu I, Gabriel R, Mangum R.   +10 more
europepmc   +2 more sources

Correction: Onasemnogene Abeparvovec for Treating Pre-symptomatic Spinal Muscular Atrophy: An External Assessment Group Perspective of the Partial Review of NICE Highly Specialised Technology Evaluation 15. [PDF]

Pharmacoecon Open, 2023
Chaplin M, Bresnahan R, Fleeman N, Mahon J, Houten R, Beale S, Boland A, Dundar Y, Marsden A, Munot P.   +9 more
europepmc   +3 more sources

Matching-adjusted indirect treatment comparison of onasemnogene abeparvovec and nusinersen for the treatment of symptomatic patients with spinal muscular atrophy type 1 [PDF]

Current Medical Research and Opinion, 2021
Matthias Bischof, Maria Lorenzi, Jennifer Lee, Eric Druyts, Chakrapani Balijepalli, Omar Dabbous   +5 more
openalex   +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

Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapies [PDF]

, 2023
Background: Efficacy and safety of onasemnogene abeparvovec (OA) for Spinal Muscular Atrophy infants under 7 months and <8.5 kg has been reported in clinical trials.
Ricci, Federica
core   +4 more sources

Onasemnogene Abeparvovec Gene Therapy and Risdiplam for the Treatment of Spinal Muscular Atrophy in Thailand: A Cost-Utility Analysis. [PDF]

Appl Health Econ Health Policy
Khuntha S, Prawjaeng J, Ponragdee K, Sanmaneechai O, Srinonprasert V, Leelahavarong P.   +5 more
europepmc   +3 more sources

Опыт применения генозаместительной терапии препаратом Золгенсма® (онасемноген абепарвовек) в реальной клинической практике в России [PDF]

, 2022
Objective: to analyze the safety and evaluate the effectiveness of therapy with onasemnogene abeparvovec in patients with spinal muscular atrophy in real clinical practice based on the experience of using the drug in the neuromuscular center of Research ...
A. V. Monakhova, D. V. Vlodavets, O. A. Shidlovskaya, S. B. Artemyeva, Yu. O. Papina, А. В. Монахова, Д. В. Влодавец, О. А. Шидловская, С. Б. Артемьева, Ю. О. Папина   +9 more
core   +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