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1.
Severe communication delays are independent of seizure burden and persist despite contemporary treatments in SCN1A+ Dravet syndrome: Insights from the ENVISION natural history study.
Epilepsia
; 65(2): 322-337, 2024 Feb.
Artículo
en Inglés
| MEDLINE | ID: mdl-38049202
2.
The clinical, economic, and humanistic burden of Dravet syndrome - A systematic literature review.
Epilepsy Behav
; 130: 108661, 2022 05.
Artículo
en Inglés
| MEDLINE | ID: mdl-35334258
3.
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).
J Mark Access Health Policy
; 9(1): 1889841, 2021 Feb 28.
Artículo
en Inglés
| MEDLINE | ID: mdl-33708361
4.
Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1.
Adv Ther
; 36(5): 1164-1176, 2019 05.
Artículo
en Inglés
| MEDLINE | ID: mdl-30879249
5.
Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients.
J Mark Access Health Policy
; 7(1): 1601484, 2019.
Artículo
en Inglés
| MEDLINE | ID: mdl-31105909
6.
Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy.
Pediatr Pulmonol
; 54(2): 179-185, 2019 02.
Artículo
en Inglés
| MEDLINE | ID: mdl-30548438
7.
Successful treatment of a patient with severe atopic dermatitis and severe asthma by centrifugal therapeutic plasma exchange.
BMJ Case Rep
; 20152015 May 12.
Artículo
en Inglés
| MEDLINE | ID: mdl-25969487
8.
Response to: Alfred Sandrock, Wildon Farwell. Letter to the Editor, Comparisons Between Separately Conducted Clinical Trials: Letter to the Editor Regarding Dabbous O, Maru B, Jansen JP, Lorenzi M, Cloutier M, Guérin A, et al. Adv Ther (2019) 36(5):1164-76. doi:10.1007/s12325-019-00923-8.
Adv Ther
; 36(11): 2982-2985, 2019 11.
Artículo
en Inglés
| MEDLINE | ID: mdl-31512141
9.
Potential use of an estrogen-glucocorticoid receptor chimera as a drug screen for tissue selective estrogenic activity.
Bone
; 44(1): 102-12, 2009 Jan.
Artículo
en Inglés
| MEDLINE | ID: mdl-18976723
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