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Unlocking the Mysteries of Rare Disease Drug Development: A Beginner's Guide for Clinical Pharmacologists.

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Title: Unlocking the Mysteries of Rare Disease Drug Development: A Beginner's Guide for Clinical Pharmacologists.
Authors: Ahmed MA; Takeda Pharmaceuticals, Cambridge, MA, USA., AbuAsal B; Boehringer Ingelheim, Ridgefield, CT, USA., Barrett JS; Aridhia Bioinformatics, Glasgow, UK., Azer K; Novartis, Cambridge, MA, USA., Hon YY; US Food and Drug Administration, Silver Spring, Maryland, USA., Albusaysi S; Department of Pharmaceutics, Faculty of Pharmacy, King Abdulaziz University, Jeddah, Saudi Arabia., Shang E; Merck & Co., Inc., Rahway, New Jersey, USA., Wang M; US Food and Drug Administration, Silver Spring, Maryland, USA., Burian M; iGenePharma Consulting, Stolberg, Germany., Rayad N; Alexion, AstraZeneca Rare Disease, Mississauga, Ontario, Canada.
Source: Clinical and translational science [Clin Transl Sci] 2025 Apr; Vol. 18 (4), pp. e70215.
Publication Type: Journal Article; Review
Journal Info: Publisher: WileyBlackwell Pub Country of Publication: United States NLM ID: 101474067 Publication Model: Print Cited Medium: Internet ISSN: 1752-8062 (Electronic) Linking ISSN: 17528054 NLM ISO Abbreviation: Clin Transl Sci Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: <searchLink fieldCode="AU" term="%22Ahmed+MA%22">Ahmed MA</searchLink>; Takeda Pharmaceuticals, Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22AbuAsal+B%22">AbuAsal B</searchLink>; Boehringer Ingelheim, Ridgefield, CT, USA.<br /><searchLink fieldCode="AU" term="%22Barrett+JS%22">Barrett JS</searchLink>; Aridhia Bioinformatics, Glasgow, UK.<br /><searchLink fieldCode="AU" term="%22Azer+K%22">Azer K</searchLink>; Novartis, Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Hon+YY%22">Hon YY</searchLink>; US Food and Drug Administration, Silver Spring, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Albusaysi+S%22">Albusaysi S</searchLink>; Department of Pharmaceutics, Faculty of Pharmacy, King Abdulaziz University, Jeddah, Saudi Arabia.<br /><searchLink fieldCode="AU" term="%22Shang+E%22">Shang E</searchLink>; Merck & Co., Inc., Rahway, New Jersey, USA.<br /><searchLink fieldCode="AU" term="%22Wang+M%22">Wang M</searchLink>; US Food and Drug Administration, Silver Spring, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Burian+M%22">Burian M</searchLink>; iGenePharma Consulting, Stolberg, Germany.<br /><searchLink fieldCode="AU" term="%22Rayad+N%22">Rayad N</searchLink>; Alexion, AstraZeneca Rare Disease, Mississauga, Ontario, Canada.
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              Text: 2025 Apr
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