Expert Opinion on Orphan Drugs, Volume 6, Issue 7 (2018)

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Expert Opinion on Orphan Drugs, Volume 6, Issue 7 (2018)

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Review

Review
Emerging therapeutic targets for primary sclerosing cholangitis
Amir Kalani, James H. Tabibian & Keith D. Lindor
Pages: 393-401
Published online: 29 Jun 2018
AbstractforEmerging therapeutic targets for primary sclerosing cholangitis | Full Text | References | PDF (862.9 KB)
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Review
Intrathecal/Intracerebroventricular enzyme replacement therapy for the mucopolysaccharidoses: efficacy, safety, and prospects
Roberto Giugliani, Amauri Dalla Corte, Fabiano Poswar, Cláudia Vanzella, Dafne Horovitz, Mariluce Riegel, Guilherme Baldo & Filippo Vairo
Pages: 403-411
Published online: 22 Jun 2018
AbstractforIntrathecal/Intracerebroventricular enzyme replacement therapy for the mucopolysaccharidoses: efficacy, safety, and prospects | Full Text | References | PDF (1.6 MB)
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Review
Advances in genetic understanding of gorlin syndrome and emerging treatment options
Shawn Shih, Christina Dai, Ahmed Ansari, Brittany A Urso, Amy I Laughlin & James A Solomon
Pages: 413-423
Published online: 14 Jun 2018
AbstractforAdvances in genetic understanding of gorlin syndrome and emerging treatment options | Full Text | References | PDF (921.9 KB)
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Review
Advances in understanding disease mechanisms and potential treatments for Crigler–Najjar syndrome
Giulia Bortolussi & Andrés Fernando Muro
Pages: 425-439
Published online: 16 Jul 2018
AbstractforAdvances in understanding disease mechanisms and potential treatments for Crigler–Najjar syndrome | Full Text | References | PDF (1.5 MB)
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Original Research

Article
Identification of approval conditions for orphan drugs for neurological disorders by the Japanese regulatory agency
Harumasa Nakamura, Shin’ichi Takeda & Masaru Iwasaki
Pages: 441-447
Published online: 11 Jul 2018
AbstractforIdentification of approval conditions for orphan drugs for neurological disorders by the Japanese regulatory agency | Full Text | References | PDF (811.5 KB)
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