Expert Opinion on Orphan Drugs, Volume 8, Issue 11 (2020)

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Expert Opinion on Orphan Drugs, Volume 8, Issue 11 (2020)

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Review

Putative adjunct therapies to target mitochondrial dysfunction and oxidative stress in phenylketonuria, lysosomal storage disorders and peroxisomal disorders
Nadia Turton, Tricia Rutherford, Dick Thijssen & Iain P Hargreaves
Pages: 431-444
Published online: 26 Nov 2020
AbstractforPutative adjunct therapies to target mitochondrial dysfunction and oxidative stress in phenylketonuria, lysosomal storage disorders and peroxisomal disorders | Full Text | References | PDF (3.5 MB) | EPUB
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Cerliponase alfa for CLN2 disease, a promising therapy
Shawn C. Aylward, Jonathan Pindrik, Nicolas J. Abreu, W. Bruce Cherny, Matthew O’Neal & Emily de Los Reyes
Pages: 445-454
Published online: 21 Dec 2020
AbstractforCerliponase alfa for CLN2 disease, a promising therapy | Full Text | References | PDF (3 MB) | EPUB
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Advances in understanding of Netherton syndrome and therapeutic implications
Evgeniya Petrova & Alain Hovnanian
Pages: 455-487
Published online: 29 Dec 2020
AbstractforAdvances in understanding of Netherton syndrome and therapeutic implications | Full Text | References | PDF (10.4 MB) | EPUB
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Open Access

Original Research

Article
Inhibitory effect of Tunceli garlic (Allium tuncelianum) on blastocystis subtype 3 grown in vitro
Mehmet Aykur, Emrah Karakavuk, Muhammet Karakavuk, Mesut Akıl, Hüseyin Can, Mert Döşkaya, Yüksel Gürüz & Hande Dağcı
Pages: 489-496
Published online: 14 Dec 2020
AbstractforInhibitory effect of Tunceli garlic (Allium tuncelianum) on blastocystis subtype 3 grown in vitro | Full Text | References | PDF (7 MB) | EPUB
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