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Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 3, Issue 5 (2015)

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Expert Opinion on Orphan Drugs, Volume 3, Issue 5 (2015)

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Editorial

Prenatal diagnosis of congenital adrenal hyperplasia owing to 21-hydroxylase deficiency

Mabel Yau MD, Christian Pina, Ahmed Khattab MD, Ariella Barhen MD & Maria I New MD

Pages: 487-490

Published online: 14 Apr 2015

AbstractforPrenatal diagnosis of congenital adrenal hyperplasia owing to 21-hydroxylase deficiency | Full Text | References | PDF (153.6 KB)

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Free Access

Review

Pathogenesis, emerging therapeutic targets and treatment in sialidosis

Alessandra D’Azzo, Eda Machado & Ida Annunziata

Pages: 491-504

Published online: 13 Apr 2015

AbstractforPathogenesis, emerging therapeutic targets and treatment in sialidosis | Full Text | References | PDF (820.8 KB)

177 Views
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Pharmacological approach to treating eosinophilic granulomatosis with polyangiitis (Churg–Strauss)

Matthieu Groh MD MSc, Bertrand Dunogué MD MSc & Loïc Guillevin MD

Pages: 505-515

Published online: 14 Apr 2015

AbstractforPharmacological approach to treating eosinophilic granulomatosis with polyangiitis (Churg–Strauss) | Full Text | References | PDF (293.1 KB)

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Machado Joseph disease: clinical and genetic aspects, and current treatment

Jonas Alex Morales Saute MD PhD & Laura Bannach Jardim (Professor) MD PhD

Pages: 517-535

Published online: 18 Mar 2015

AbstractforMachado Joseph disease: clinical and genetic aspects, and current treatment | Full Text | References | PDF (787.1 KB)

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Phase II clinical trials for Waldenstrom’s macroglobulinemia

Rajshekhar Chakraborty, Stephen A Ansell, Prashant Kapoor & Morie A Gertz MD MACP

Pages: 537-547

Published online: 01 Apr 2015

AbstractforPhase II clinical trials for Waldenstrom’s macroglobulinemia | Full Text | References | PDF (231 KB)

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Understanding and managing parkinsonism in patients with glucocerebrosidase mutations

Jenny Kim, Ellen Sidransky MD & Grisel Lopez MD

Pages: 549-562

Published online: 23 Apr 2015

AbstractforUnderstanding and managing parkinsonism in patients with glucocerebrosidase mutations | Full Text | References | PDF (322.3 KB)

81 Views
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Improvement in vision: a new goal for treatment of hereditary retinal degenerations

Samuel G Jacobson MD PhD, Artur V Cideciyan PhD, Gustavo D Aguirre VMD PhD, Alejandro J Roman MS, Alexander Sumaroka PhD, William W Hauswirth PhD & Krzysztof Palczewski PhD

Pages: 563-575

Published online: 26 Mar 2015

AbstractforImprovement in vision: a new goal for treatment of hereditary retinal degenerations | Full Text | References | PDF (752.3 KB)

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Open Access

Mucosa-associated lymphoid tissue lymphoma: a perspective on current and future therapeutic strategies

Barbara Kiesewetter & Markus Raderer MD

Pages: 577-586

Published online: 08 Apr 2015

AbstractforMucosa-associated lymphoid tissue lymphoma: a perspective on current and future therapeutic strategies | Full Text | References | PDF (413.5 KB)

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Drug Evaluation

Pirfenidone: an orphan drug for treating idiopathic pulmonary fibrosis

Yukiko Miura & Arata Azuma

Pages: 587-597

Published online: 17 Apr 2015

AbstractforPirfenidone: an orphan drug for treating idiopathic pulmonary fibrosis | Full Text | References | PDF (600.2 KB)

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Bosutinib for the treatment of Philadelphia chromosome-positive leukemias

Cristina Varallo-Rodriguez, Craig W Freyer, Evelena P Ontiveros, Elizabeth A Griffiths, Eunice S Wang & Meir Wetzler

Pages: 599-608

Published online: 16 Apr 2015

AbstractforBosutinib for the treatment of Philadelphia chromosome-positive leukemias | Full Text | References | PDF (264 KB)

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Brentuximab vedotin for the treatment of Hodgkin’s and non-Hodgkin’s lymphoma

Patrick M Reagan & Jonathan W Friedberg

Pages: 609-618

Published online: 14 Apr 2015

AbstractforBrentuximab vedotin for the treatment of Hodgkin’s and non-Hodgkin’s lymphoma | Full Text | References | PDF (384.5 KB)

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Expert Opinion on Orphan Drugs (Vol.3, Iss.5, 2015)

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