المؤلفون: Bruce Wang, Paolo Ventura, Kei-ichiro Takase, Manish Thapar, David Cassiman, Ilja Kubisch, Shangbin Liu, Marianne T. Sweetser, Manisha Balwani

المصدر: Orphanet Journal of Rare Diseases, Vol 17, Iss 1, Pp 1-12 (2022)

مصطلحات موضوعية: Acute hepatic porphyria, Givosiran, Disease burden, Porphyria attack, Chronic symptoms, Quality of life, Medicine

وصف الملف: electronic resource

العلاقة: https://doaj.org/toc/1750-1172Test

الوصول الحر: https://doaj.org/article/126628ee581049f09f0b982642040408Test

المؤلفون: Laurent Gouya, D. Montgomery Bissell, David J. Kuter, Marianne T. Sweetser, Herbert L. Bonkovsky, Paolo Ventura, Paula Aguilera Peiró, Eliane Sardh, Karl E. Anderson, Jeeyoung Oh, Penelope E. Stein, Zhaowei Hua, John J. Ko, Susana Monroy

المصدر: American Journal of Gastroenterology. 116:S545-S546

مصطلحات موضوعية: Acute hepatic porphyria, medicine.medical_specialty, Hepatology, Randomized controlled trial, business.industry, law, Internal medicine, Gastroenterology, Medicine, In patient, business, Interim analysis, law.invention

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::7211b2ec87cf0d20182a2ccd4827c613Test
https://doi.org/10.14309/01.ajg.0000778212.30789.aeTest

المؤلفون: Bruce Wang, Paolo Ventura, Kei-ichiro Takase, Manish Thapar, David Cassiman, Ilja Kubisch, Shangbin Liu, Marianne T. Sweetser, Manisha Balwani

المصدر: Orphanet Journal of Rare Diseases, vol 17, iss 1

مصطلحات موضوعية: Acute Porphyrias, Acute hepatic porphyria, Givosiran, Disease burden, Porphyria attack, Chronic symptoms, Quality of life, Porphobilinogen, Neurodegenerative, Research & Experimental Medicine, ACUTE INTERMITTENT PORPHYRIA, Hepatic, Cost of Illness, Envision, Pharmacology (medical), Genetics (clinical), Burden, Acute Porphyrias, Envision, Genetics & Heredity, Pain Research, PAIN, Hematology, Disease burden, General Medicine, LIVER-TRANSPLANTATION, Porphyria attack, Medicine, Research & Experimental, 6.1 Pharmaceuticals, Hemin, Chronic Pain, Life Sciences & Biomedicine, Quality of life, Clinical Trials and Supportive Activities, Pain, Burden, Porphyrias, Clinical Research, Acute hepatic porphyria, Genetics, MANAGEMENT, Humans, Peripheral Neuropathy, Givosiran, Other Medical and Health Sciences, Science & Technology, Porphyria, Chronic symptoms, Neurosciences, Evaluation of treatments and therapeutic interventions, Porphobilinogen Synthase, Acute Intermittent, Porphyrias, Hepatic, Porphyria, Acute Intermittent, Quality of Life, UPDATE, Digestive Diseases, ATTACKS

وصف الملف: Electronic; application/pdf

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d3dcc4a34a20afd30da2fd2112dd67d6Test
https://hdl.handle.net/11380/1261337Test

المؤلفون: Herbert L. Bonkovsky, Ming-Jen Lee, David Cassiman, Aneta Ivanova, Susana Monroy, Encarna Guillen-Navarro, Marianne T. Sweetser, Zhaowei Hua, Facg, Manish Thapar, Raili M. Kauppinen

المصدر: American Journal of Gastroenterology. 116:S546-S546

مصطلحات موضوعية: Acute hepatic porphyria, Pediatrics, medicine.medical_specialty, Hepatology, business.industry, Gastroenterology, medicine, business, Natural history study, Term (time)

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::b50c061b5e4f4a12628fbc3267855a2bTest
https://doi.org/10.14309/01.ajg.0000778216.45955.7fTest

المؤلفون: Paolo Ventura, Bruce Wang, Manisha Balwani, Marianne T. Sweetser, Manish Thapar, Zhaowei Hua, Kei-ichiro Takase, Ilja Kubisch, David Cassiman

المصدر: American Journal of Gastroenterology. 116:S504-S504

مصطلحات موضوعية: Acute hepatic porphyria, medicine.medical_specialty, Hepatology, business.industry, Internal medicine, Gastroenterology, medicine, In patient, business, Disease burden

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::4edf2caa72042a5c57b9479b23d941c8Test
https://doi.org/10.14309/01.ajg.0000777784.56221.d2Test