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. |
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| 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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| ISSN: | 1752-8062 |
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| DOI: | 10.1111/cts.70215 |
