Literature DB >> 27054988

Accessing the accelerated approval pathway for rare disease therapeutics.

Emil D Kakkis1, Sara Kowalcyk1, Max G Bronstein1.   

Abstract

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Year:  2016        PMID： 27054988     DOI： 10.1038/nbt.3530

Source DB:  PubMed          Journal:  Nat Biotechnol        ISSN： 1087-0156            Impact factor:   54.908


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  3 in total

1.  Phenylalanine blood levels and clinical outcomes in phenylketonuria: a systematic literature review and meta-analysis.

Authors:  Susan E Waisbren; Kay Noel; Kyle Fahrbach; Catherine Cella; Diana Frame; Alex Dorenbaum; Harvey Levy
Journal:  Mol Genet Metab       Date:  2007-06-25       Impact factor: 4.797

2.  The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseases.

Authors:  Brigitta E Miyamoto; Emil D Kakkis
Journal:  Orphanet J Rare Dis       Date:  2011-07-06       Impact factor: 4.123

Review 3.  Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints.

Authors:  Emil D Kakkis; Mary O'Donovan; Gerald Cox; Mark Hayes; Federico Goodsaid; P K Tandon; Pat Furlong; Susan Boynton; Mladen Bozic; May Orfali; Mark Thornton
Journal:  Orphanet J Rare Dis       Date:  2015-02-10       Impact factor: 4.123
  3 in total
  1 in total

1.  Use of antisense oligonucleotides to correct the splicing error in ISCU myopathy patient cell lines.

Authors:  Gregory P Holmes-Hampton; Daniel R Crooks; Ronald G Haller; Shuling Guo; Susan M Freier; Brett P Monia; Tracey A Rouault
Journal:  Hum Mol Genet       Date:  2016-12-01       Impact factor: 6.150
  1 in total

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