نتائج البحث

Clinical features of acute attacks, chronic symptoms, and long-term complications among patients with acute hepatic porphyria in Japan: a real-world claims database study.

• Authors : Horie Y; Department of Gastroenterology, Saiseikai Gotsu General Hospital, Shimane, Japan.; Yasuoka Y

Subjects: Porphyrias, Hepatic*/Porphyrias, Hepatic*/Porphyrias, Hepatic*/complications ; Porphyrias, Hepatic*/Porphyrias, Hepatic*/Porphyrias, Hepatic*/epidemiology ; Porphyria, Acute Intermittent*/Porphyria, Acute Intermittent*/Porphyria, Acute Intermittent*/complications Porphyria, Acute Hepatic

• Source: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2023 Dec 08; Vol. 18 (1), pp. 384. Date of Electronic Publication: 2023 Dec 08.Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172

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A high urinary urobilinogen/serum total bilirubin ratio indicates acute hepatic porphyria in patients with abdominal pain.

• Authors : Song C; Department of Neurology, Qilu Hospital of Shandong University, Jinan, 250012, People's Republic of China.; Liu Y

Subjects: Urobilinogen*/Urobilinogen*/Urobilinogen*/urine ; Porphyrias, Hepatic*; Humans Porphyria, Acute Hepatic

• Source: Scientific reports [Sci Rep] 2023 Dec 04; Vol. 13 (1), pp. 21330. Date of Electronic Publication: 2023 Dec 04.Publisher: Nature Publishing Group Country of Publication: England NLM ID: 101563288 Publication Model: Electronic Cited Medium: Internet ISSN:

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Small interfering RNA: Discovery, pharmacology and clinical development-An introductory review.

• Authors : Ranasinghe P; Department of Pharmacology, Faculty of Medicine, University of Colombo, Colombo, Sri Lanka.; University/British Heart Foundation Centre for Cardiovascular Science, The University of Edinburgh, Edinburgh, UK.

Subjects: RNA, Double-Stranded* ; Porphyrias, Hepatic*/Porphyrias, Hepatic*/Porphyrias, Hepatic*/drug therapy ; Porphyrias, Hepatic*/Porphyrias, Hepatic*/Porphyrias, Hepatic*/genetics

• Source: British journal of pharmacology [Br J Pharmacol] 2023 Nov; Vol. 180 (21), pp. 2697-2720. Date of Electronic Publication: 2022 Nov 13.Publisher: Wiley Country of Publication: England NLM ID: 7502536 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1476-5381

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Pharmacokinetic-pharmacodynamic model of urinary δ-aminolevulinic acid reduction after givosiran treatment in patients with acute hepatic porphyria.

• Authors : Lee J; Alnylam Pharmaceuticals, Cambridge, Massachusetts, USA.; Melch M

Subjects: Aminolevulinic Acid* ; Porphyrias, Hepatic*/Porphyrias, Hepatic*/Porphyrias, Hepatic*/drug therapy; Adult Porphyria, Acute Hepatic

• Source: CPT: pharmacometrics & systems pharmacology [CPT Pharmacometrics Syst Pharmacol] 2023 Jun; Vol. 12 (6), pp. 842-852. Date of Electronic Publication: 2023 Mar 23.Publisher: Wiley Country of Publication: United States NLM ID: 101580011 Publication Model: Print-Electronic Cited Medium: Internet ISSN:

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ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP) (ELEVATE)

• Source: ELEVATE, a Global Observational Longitudinal Prospective Registry of Patients With Acute Hepatic Porphyria (AHP)

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Evaluating the Prevalence of Acute Hepatic Porphyria in Postural Tachycardia Syndrome

• Authors : Cyndya Shibao, MD, Associate Professor

• Source: Estimating the Frequency and Neuro-Hormonal Characteristics of Acute Hepatic Porphyria in Postural Tachycardia SyndromeRobertson D. The epidemic of orthostatic

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Challenges in diagnosis and management of acute hepatic porphyrias: from an uncommon pediatric onset to innovative treatments and perspectives.

• Authors : Marcacci M; Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Via del Pozzo 71, 41124, Modena, Italy.; Ricci A

Subjects: Porphyria, Acute Intermittent*/Porphyria, Acute Intermittent*/Porphyria, Acute Intermittent*/diagnosis ; Porphyria, Acute Intermittent*/Porphyria, Acute Intermittent*/Porphyria, Acute Intermittent*/therapy ; Porphyrias*/Porphyrias*/Porphyrias*/complications Porphyria, Acute Hepatic

• Source: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2022 Apr 07; Vol. 17 (1), pp. 160. Date of Electronic Publication: 2022 Apr 07.Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172

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Disease burden in patients with acute hepatic porphyria: experience from the phase 3 ENVISION study.

• Authors : Wang B; Division of Gastroenterology, Department of Medicine, University of California San Francisco, San Francisco, CA, 94143, USA. .; Ventura P

Subjects: Porphyria, Acute Intermittent* ; Porphyrias, Hepatic*; Cost of Illness Porphyria, Acute Hepatic

• Source: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2022 Aug 26; Vol. 17 (1), pp. 327. Date of Electronic Publication: 2022 Aug 26.Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172

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Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria

• Source: Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria (AHP)

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ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)

• Source: ENVISION: A Phase 3 Randomized, Double-blind, Placebo-Controlled Multicenter Study With an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients With Acute

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