Found: 11
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Concept confirmation of the Treatment Administration Satisfaction Questionnaire (TASQ) in rare paroxysmal nocturnal hemoglobinuria.
- Published in:
- Journal of Patient-Reported Outcomes, 2021, v. 5, n. 1, p. 1, doi. 10.1186/s41687-021-00319-9
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- Publication type:
- Article
Assessing Preferences for Rare Disease Treatment: Qualitative Development of the Paroxysmal Nocturnal Hemoglobinuria Patient Preference Questionnaire (PNH-PPQ©).
- Published in:
- Patient Preference & Adherence, 2020, v. 14, p. 705, doi. 10.2147/PPA.S233830
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- Publication type:
- Article
Frequency and age at occurrence of clinical manifestations of disease in patients with hypophosphatasia: a systematic literature review.
- Published in:
- 2019
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- Publication type:
- journal article
Predictors for improvement in patient-reported outcomes: post hoc analysis of a phase 3 randomized, open-label study of eculizumab and ravulizumab in complement inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria.
- Published in:
- Annals of Hematology, 2024, v. 103, n. 1, p. 5, doi. 10.1007/s00277-023-05483-0
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- Publication type:
- Article
The burden of mitochondrial disease with associated seizures: systematic literature reviews of health-related quality of life, utilities, costs and healthcare resource use data.
- Published in:
- Orphanet Journal of Rare Diseases, 2023, v. 18, n. 1, p. 1, doi. 10.1186/s13023-023-02945-6
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- Publication type:
- Article
Patient preferences and quality of life implications of ravulizumab (every 8 weeks) and eculizumab (every 2 weeks) for the treatment of paroxysmal nocturnal hemoglobinuria.
- Published in:
- PLoS ONE, 2020, v. 15, n. 9, p. 1, doi. 10.1371/journal.pone.0237497
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- Publication type:
- Article
Clinically important change for the FACIT-Fatigue scale in paroxysmal nocturnal hemoglobinuria: a derivation from international PNH registry patient data.
- Published in:
- Journal of Patient-Reported Outcomes, 2023, v. 7, n. 1, p. 1, doi. 10.1186/s41687-023-00609-4
- By:
- Publication type:
- Article
Cost-Utility Analysis of Ravulizumab Compared with Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria.
- Published in:
- PharmacoEconomics, 2020, v. 38, n. 9, p. 981, doi. 10.1007/s40273-020-00929-z
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- Publication type:
- Article
Predictors of cardiac disease in duchenne muscular dystrophy: a systematic review and evidence grading.
- Published in:
- Orphanet Journal of Rare Diseases, 2024, v. 19, n. 1, p. 1, doi. 10.1186/s13023-024-03372-x
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- Publication type:
- Article
Low Rate of Clinically Evident Extravascular Hemolysis in Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with a Complement C5 Inhibitor: Results from a Large, Multicenter, US Real-World Study.
- Published in:
- Journal of Blood Medicine, 2022, v. 13, p. 425, doi. 10.2147/JBM.S361863
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- Publication type:
- Article
Reliability and Validity of the 6‐Minute Walk Test in Hypophosphatasia.
- Published in:
- JBMR Plus, 2019, v. 3, n. 6, p. N.PAG, doi. 10.1002/jbm4.10131
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- Publication type:
- Article