Found: 13
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Use of the patient-reported outcomes measurement information system (PROMIS®) to assess late-onset Pompe disease severity.
- Published in:
- Journal of Patient-Reported Outcomes, 2020, v. 4, n. 1, p. N.PAG, doi. 10.1186/s41687-020-00245-2
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- Publication type:
- Article
Regulatory strategies for rare diseases under current global regulatory statutes: a discussion with stakeholders.
- Published in:
- 2019
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- Publication type:
- journal article
FABry Disease Patient-Reported Outcome-GastroIntestinal (FABPRO-GI): A new Fabry disease-specific gastrointestinal outcomes instrument.
- Published in:
- Quality of Life Research, 2021, v. 30, n. 10, p. 2983, doi. 10.1007/s11136-021-02847-9
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- Article
Intestinal cytokines in children with pervasive developmental disorders
- Published in:
- American Journal of Gastroenterology (Springer Nature), 2003, v. 98, n. 8, p. 1777, doi. 10.1111/j.1572-0241.2003.07593.x
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- Publication type:
- Article
Extrapolation of Adult Efficacy to Pediatric Patients With Chemotherapy‐Induced Nausea and Vomiting.
- Published in:
- Journal of Clinical Pharmacology, 2020, v. 60, n. 6, p. 775, doi. 10.1002/jcph.1577
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- Publication type:
- Article
Pediatric Intestinal Failure-Associated Liver Disease: Challenges in Identifying Clinically Relevant Biomarkers.
- Published in:
- 2018
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- Publication type:
- journal article
Proceedings From FDA/A.S.P.E.N. Public Workshop.
- Published in:
- JPEN Journal of Parenteral & Enteral Nutrition, 2015, v. 39, n. 7, p. 768, doi. 10.1177/0148607114560825
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- Publication type:
- Article
Turnover of the cystic fibrosis transmembrane conductance regulator (CFTR): Slow degradation of wild-type and ΔF508 CFTR in surface membrane preparations of immortalized airway epithelial cells.
- Published in:
- Journal of Cellular Physiology, 1996, v. 168, n. 2, p. 373, doi. 10.1002/(SICI)1097-4652(199608)168:2<373::AID-JCP16>3.0.CO;2-4
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- Publication type:
- Article
Reduced expression of the cystic fibrosis transmembrane conductance regulator gene in the hypothalamus of patients with Alzheimer's disease.
- Published in:
- Journal of Alzheimer's Disease, 2003, v. 5, n. 6, p. 455
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- Publication type:
- Article
Exposure Matching for Extrapolation of Efficacy in Pediatric Drug Development.
- Published in:
- Journal of Clinical Pharmacology, 2016, v. 56, n. 11, p. 1326, doi. 10.1002/jcph.744
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- Publication type:
- Article
Data silos are undermining drug development and failing rare disease patients.
- Published in:
- 2021
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- Publication type:
- journal article
Correction to: Sharing is caring: a call for a new era of rare disease research and development.
- Published in:
- Orphanet Journal of Rare Diseases, 2023, v. 18, n. 1, p. 1, doi. 10.1186/s13023-023-02613-9
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- Publication type:
- Article
Sharing is caring: a call for a new era of rare disease research and development.
- Published in:
- 2022
- By:
- Publication type:
- journal article