Nutritional Treatment for Inborn Errors of Metabolism: Indications, Regulations, and Availability of Medical Foods and Dietary Supplements Using Phenylketonuria as an Example, Kathryn M. Campa,*, Michele A. Lloyd-Puryearb, and Kathleen L. Huntington Mol Genet Metab. 2012 September ; 107(1-2): 3–9. doi:10.1016/j.ymgme.2012.07.005.
Pharmacotherapy of inborn errors of metabolism illustrating challenges in orphan diseases Anibh M. Das http://dx.doi.org/10.1016/j.vascn.2016.02.182, 1056-8719/© 2016 Elsevier Inc. All rights reserved.
Gambello, M.J., Li, H., Current strategies for the treatment of inborn errors of metabolism, Journal of Genetics and Genomics (2018), doi: 10.1016/j.jgg.2018.02.001.
Accessing the accelerated approval pathway for rare disease therapeutics, Emil D Kakkis, Sara Kowalcyk & Max G Bronstein, Improvements must be made to the qualification process for biomarkers as primary endpoints in pivotal clinical studies of treatments for the rarest of diseases. VOLUME 34 NUMBER 4 APRIL 2016 NATURE BIOTECHNOLOGY
Replacement Therapies in Metabolic Disease, Alexander S. Keller1,2, T.C. Stevenson Keller IV1,3, Leon J. DeLalio1,2, Guleer Shahab1, Yang Yang4,5, Sara A. Murphy1, Xiaohong Shu4,5 and Brant E. Isakson1,3*DOI:10.2174/1389201019666180619151413 Current Pharmaceutical Biotechnology, 2018, 19, 382-399
Advocates Call for Long-Term Extension of the FDA’s Rare Pediatric, Disease Priority Review Program Federal voucher program considered instrumental in shortening drug review, approval period, DOI: 10.1002/ajmg.a.38091 2017 Wiley Periodicals, Inc.
David B. Ridley and Stephane A. Régnier, The Commercial Market For Priority Review Vouchers, doi: 10.1377/hlthaff.2015.1314 HEALTH AFFAIRS 35, NO. 5 (2016): 776–783©2016 Project HOPE—The People-to-People Health Foundation, Inc.
Streamlining nonclinical drug development using the FDA 505(b)(2) new drug application regulatory pathway William F. Salminen, Marc E.Wiles, Ruth E. Stevens PII: S1359-6446(18)30120-X DOI: https://doi.org/10.1016/j.drudis.2018.07.005, Reference: DRUDIS 2288
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