| Real-world Assessment of Renal Involvement in Tuberous Sclerosis Complex (TSC) Patients in the United Kingdom (UK): A Retrospective Cohort Study in the Clinical Practice Research Datalink (CPRD) |
| Author(s): Kingswood, Chris1, Nasuti, Paola2, Demuth, Dirk2, Patel, Keyur2, Myland, Melissa2, Erhard, Clement3, Gray, Elizabeth4, Pinnegar, Alun4, Magestro, Matthew5 |
| Affiliations(s): The Royal Sussex County Hospital, Brighton, England
2 IMS Health, London, UK
3 CE Analytics, Cambridge, UK
4 Novartis Pharmaceuticals UK Ltd, Frimley, UK
5 Novartis Pharmaceuticals Corporation, East Hanover NJ, USA
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| Publication(s): International TSC Research Conference (Windsor, 11th Sept 2015). |
| Document Type(s): Poster |
| Countries: England |
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Oncology, Pediatrics, Rare disease
2015 |
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English
Retrospective cohort analysis |
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| Association of sweat chloride concentration at time of diagnosis and CFTR genotype with mortality and cystic fibrosis phenotype |
| Author(s): McKone EF, Goss C, Velentgas P, Swenson A |
| Affiliations(s): |
| Publication(s): Journal of Cystic Fibrosis. JCF1155. Feb 2015. 10.1016(j.jcf.2015) 01.005 |
| Document Type(s): Article |
| Countries: |
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Rare disease
2015 |
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English
RWLPR (Real-World Late Phase Research-quintiles |
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| Fifth interim analysis of the Kuvan® Adult Maternal Paediatric European Registry (KAMPER): Insights into management/practice in patients with PKU. |
| Author(s): Lagler FB, Muntau AC, Feillet F, Rogoff D, Moreau-Stucker F, Alm J, Burlina A, Trefz FK. |
| Affiliations(s): Quintiles Real-World and Late Phase Research |
| Publication(s): Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Congress, 1–4 September 2015, Lyon, France, Abstract No. P-160 |
| Document Type(s): Abstract |
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Patient Registries & Real-World Study Methods, Rare disease
2015 |
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English
RWLPR (Real-World Late Phase Research-quintiles |
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| Impact of Acute Bleeding on Daily Activities of Patients with Congenital Hemophilia with Inhibitors and Their Caregivers and Families: Observations from the Dosing Observational Study in Hemophilia (DOSE). Science Direct |
| Author(s): Recht N, Neufeld EJ, Sharma VR, Solem CT, Pickard S, Gut RZ, MD, Cooper DL |
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| Publication(s): |
| Document Type(s): Article |
| Countries: |
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Rare disease
2014 |
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English
RWLPR (Real-World Late Phase Research-quintiles |
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| Health-related Quality of Life in Patients with Hemophilia and Inhibitors on Prophylaxis with an Activated Prothrombin Complex Concentrate: Results From the Pro-FEIBA Study |
| Author(s): Gringeri A, Leissinger C,,Cortesi P, Jo H, Fusco F, Riva F, Bulent A, Berntorp E, Carpenter S, Kavakli K, Morfini M, Negrier C, Rocino A, Schramm W, Uscatescu M, Windyga J, Zulfikar B, Mantovani LG |
| Affiliations(s): |
| Publication(s): Haemophilia 2013 Sep;19(5):736-43 |
| Document Type(s): Article |
| Countries: |
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Rare disease
2013 |
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English
RWLPR (Real-World Late Phase Research-quintiles |
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| Impact of pregnancy on women with cystic fibrosis |
| Author(s): McMullen AH, Pasta DJ, Frederick PD, Konstan MW, Morgan WJ, Schechter MS, Wagener JS |
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| Publication(s): Chest 2006; 129(3):706-711 |
| Document Type(s): Article |
| Countries: |
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Rare disease
2006 |
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English
RWLPR (Real-World Late Phase Research-quintiles |
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