Eugene Schneider
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Explore the profile of Eugene Schneider including associated specialties, affiliations and a list of published articles.
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22
Citations
2417
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Recent Articles
11.
Viney N, Guo S, Tai L, Baker B, Aghajan M, Jung S, et al.
ESC Heart Fail
. 2020 Dec;
8(1):652-661.
PMID: 33283485
Aims: Amyloidogenic transthyretin (ATTR) amyloidosis is a fatal disease characterized by progressive cardiomyopathy and/or polyneuropathy. AKCEA-TTR-L (ION-682884) is a ligand-conjugated antisense drug designed for receptor-mediated uptake by hepatocytes, the primary...
12.
Cohn D, Viney N, Fijen L, Schneider E, Alexander V, Xia S, et al.
N Engl J Med
. 2020 Sep;
383(13):1242-1247.
PMID: 32877578
Hereditary angioedema is characterized by recurrent and unpredictable episodes of subcutaneous and mucosal swelling that can be life threatening. IONIS-PKK-L is a ligand-conjugated antisense oligonucleotide designed for receptor-mediated delivery to...
13.
Narayanan P, Curtis B, Shen L, Schneider E, Tami J, Paz S, et al.
Nucleic Acid Ther
. 2020 Feb;
30(2):94-103.
PMID: 32043907
Inotersen, a 2'-O-methoxyethyl (2'-MOE) phosphorothioate antisense oligonucleotide, reduced disease progression and improved quality of life in patients with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) in the NEURO-TTR and NEURO-TTR open-label...
14.
Montes J, Dunaway Young S, Mazzone E, Pasternak A, Glanzman A, Finkel R, et al.
Muscle Nerve
. 2019 Jul;
60(4):409-414.
PMID: 31298747
Introduction: Ambulatory individuals with spinal muscular atrophy (SMA) experience muscle weakness, gait impairments, and fatigue that affect their walking ability. Improvements have been observed in motor function in children treated...
15.
Darras B, Chiriboga C, Iannaccone S, Swoboda K, Montes J, Mignon L, et al.
Neurology
. 2019 Apr;
92(21):e2492-e2506.
PMID: 31019106
Objective: To report results of intrathecal nusinersen in children with later-onset spinal muscular atrophy (SMA). Methods: Analyses included children from a phase 1b/2a study (ISIS-396443-CS2; NCT01703988) who first received nusinersen...
16.
Ferrone J, Bhattacharjee G, Revenko A, Zanardi T, Warren M, Derosier F, et al.
Nucleic Acid Ther
. 2019 Mar;
29(2):82-91.
PMID: 30817230
Kallikrein is the key contact system mediator responsible for the conversion of high-molecular-weight kininogen into the inflammatory vasodilator peptide bradykinin, a process regulated by C1-esterase inhibitor (C1-INH). In hereditary angioedema...
17.
Mercuri E, Darras B, Chiriboga C, Day J, Campbell C, Connolly A, et al.
N Engl J Med
. 2018 Feb;
378(7):625-635.
PMID: 29443664
Background: Nusinersen is an antisense oligonucleotide drug that modulates pre-messenger RNA splicing of the survival motor neuron 2 ( SMN2) gene. It has been developed for the treatment of spinal...
18.
Finkel R, Mercuri E, Darras B, Connolly A, Kuntz N, Kirschner J, et al.
N Engl J Med
. 2017 Nov;
377(18):1723-1732.
PMID: 29091570
Background: Spinal muscular atrophy is an autosomal recessive neuromuscular disorder that is caused by an insufficient level of survival motor neuron (SMN) protein. Nusinersen is an antisense oligonucleotide drug that...
19.
Finkel R, Chiriboga C, Vajsar J, Day J, Montes J, De Vivo D, et al.
Lancet
. 2016 Dec;
388(10063):3017-3026.
PMID: 27939059
Background: Nusinersen is a 2'-O-methoxyethyl phosphorothioate-modified antisense drug being developed to treat spinal muscular atrophy. Nusinersen is specifically designed to alter splicing of SMN2 pre-mRNA and thus increase the amount...
20.
Jones S, Valayannopoulos V, Schneider E, Eckert S, Banikazemi M, Bialer M, et al.
Genet Med
. 2015 Aug;
18(5):452-8.
PMID: 26312827
Purpose: The purpose of this study was to enhance understanding of lysosomal acid lipase deficiency (LALD) in infancy. Methods: Investigators reviewed medical records of infants with LALD and summarized data...