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The 21st Century Cures Act.
N Engl J Med. 2015 Oct 22;373(17):1678. doi: 10.1056/NEJMc1509640.
N Engl J Med. 2015.
PMID: 26488712
No abstract available.
Accessing the accelerated approval pathway for rare disease therapeutics.
Kakkis ED, Kowalcyk S, Bronstein MG.
Kakkis ED, et al. Among authors: bronstein mg.
Nat Biotechnol. 2016 Apr;34(4):380-3. doi: 10.1038/nbt.3530.
Nat Biotechnol. 2016.
PMID: 27054988
No abstract available.
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Patients as key partners in rare disease drug development.
Bronstein MG, Kakkis ED.
Bronstein MG, et al.
Nat Rev Drug Discov. 2016 Nov;15(11):731-732. doi: 10.1038/nrd.2016.133. Epub 2016 Jul 22.
Nat Rev Drug Discov. 2016.
PMID: 27444223
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Leveraging Evidence-Based Public Policy and Advocacy to Advance Newborn Screening in California.
Bronstein MG, Pan RJ, Dant M, Lubin B.
Bronstein MG, et al.
Pediatrics. 2019 Feb;143(2):e20181886. doi: 10.1542/peds.2018-1886. Epub 2019 Jan 3.
Pediatrics. 2019.
PMID: 30606745
Review.
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