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Eugene Schneider

ionis pharmaceuticals, inc
H指数:20
被引数:6,463

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A phase 2 open-label extension study of prekallikrein inhibition with donidalorsen for hereditary angioedema

donidalorsen对遗传性血管性水肿的前激肽释放酶抑制的2期开放标签扩展研究

2023-11-275
OA
Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy

Eplontersen用于遗传性甲状腺素蛋白淀粉样变性伴多发性神经病

2023-10-1744
OA
Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen

Eplontersen的开放标签3期研究neuro-ttransform中遗传性甲状腺素蛋白淀粉样变性多发性神经病 (attrv-pn) 患者的特征

2022-12-1626
OAAI
Inhibition of Prekallikrein for Hereditary Angioedema
2022-03-1743
OA
Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-LRx (ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
2021-02-2642
OAAI
Nusinersen in later-onset spinalmuscular atrophy Long-term results from the phase 1/2 studies
2019-05-21204
OAAI
Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy
2018-02-15970
OAAI
Rapid progression and mortality of lysosomal acid lipase deficiency presenting in infants
2016-05-0161
OAAI
Sebelipase alfa over 52 weeks reduces serum transaminases, liver volume and improves serum lipids in patients with lysosomal acid lipase deficiency
2014-11-0174
OAAI
Severe and rapid disease course in the natural history of infants with lysosomal acid lipase deficiency
2014-02-016
PREAI