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1.
Long-term survival outcomes of patients with Niemann-Pick disease type C receiving miglustat treatment: A large retrospective observational study.
J Inherit Metab Dis
; 43(5): 1060-1069, 2020 09.
Artículo
en Inglés
| MEDLINE | ID: mdl-32324281
2.
Elucidating the burden of recurrent and chronic digital ulcers in systemic sclerosis: long-term results from the DUO Registry.
Ann Rheum Dis
; 75(10): 1770-6, 2016 Oct.
Artículo
en Inglés
| MEDLINE | ID: mdl-26612339
3.
Functional impairment of systemic scleroderma patients with digital ulcerations: results from the DUO Registry.
Clin Exp Rheumatol
; 31(2 Suppl 76): 71-80, 2013.
Artículo
en Inglés
| MEDLINE | ID: mdl-23910613
4.
Demographic, clinical and antibody characteristics of patients with digital ulcers in systemic sclerosis: data from the DUO Registry.
Ann Rheum Dis
; 71(5): 718-21, 2012 May.
Artículo
en Inglés
| MEDLINE | ID: mdl-22247218
5.
Clinical characteristics and predictors of gangrene in patients with systemic sclerosis and digital ulcers in the Digital Ulcer Outcome Registry: a prospective, observational cohort.
Ann Rheum Dis
; 75(9): 1736-40, 2016 09.
Artículo
en Inglés
| MEDLINE | ID: mdl-27353738
6.
Miglustat (Zavesca) in type 1 Gaucher disease: 5-year results of a post-authorisation safety surveillance programme.
Pharmacoepidemiol Drug Saf
; 18(9): 770-7, 2009 Sep.
Artículo
en Inglés
| MEDLINE | ID: mdl-19507165
7.
Psychiatric and neurological symptoms in patients with Niemann-Pick disease type C (NP-C): Findings from the International NPC Registry.
World J Biol Psychiatry
; 20(4): 310-319, 2019 04.
Artículo
en Inglés
| MEDLINE | ID: mdl-28914127
8.
Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: an observational cohort study.
Orphanet J Rare Dis
; 10: 65, 2015 May 28.
Artículo
en Inglés
| MEDLINE | ID: mdl-26017010
9.
Disease and patient characteristics in NP-C patients: findings from an international disease registry.
Orphanet J Rare Dis
; 8: 12, 2013 Jan 16.
Artículo
en Inglés
| MEDLINE | ID: mdl-23324478
10.
Safety experience with bosentan in 146 children 2-11 years old with pulmonary arterial hypertension: results from the European Postmarketing Surveillance program.
Pediatr Res
; 64(2): 200-4, 2008 Aug.
Artículo
en Inglés
| MEDLINE | ID: mdl-18414142
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