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. Nature Biotechnology 2016;34(4).
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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