Expert Opinion on Orphan Drugs, Volume 10, Issue 1 (2022)

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Expert Opinion on Orphan Drugs, Volume 10, Issue 1 (2022)

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Review

Review
Molecular aspects of the altered Angiotensin II signaling in Gitelman’s syndrome
Verdiana Ravarotto, Giovanni Bertoldi, Lucia Federica Stefanelli, Laura Gobbi & Lorenzo A. Calò
Pages: 1-10
Published online: 03 May 2022
AbstractforMolecular aspects of the altered Angiotensin II signaling in Gitelman’s syndrome | Full Text | References | PDF (2.1 MB) | EPUB
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Review
Recommendations for overcoming challenges in the diagnosis of lysosomal acid lipase deficiency
Pilar Giraldo, Laura López de Frutos & Jorge J Cebolla
Pages: 11-21
Published online: 12 Oct 2022
AbstractforRecommendations for overcoming challenges in the diagnosis of lysosomal acid lipase deficiency | Full Text | References | PDF (570.5 KB) | EPUB
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Original Research

Article
Pricing and reimbursement policy for new orphan drugs in South Korea: focused on patient accessibility and budget impact
Jong Hyuk Lee & Seung-Lai Yoo
Pages: 22-27
Published online: 14 Oct 2022
AbstractforPricing and reimbursement policy for new orphan drugs in South Korea: focused on patient accessibility and budget impact | Full Text | References | PDF (701.5 KB) | EPUB
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Editorial

Editorial
Rare diseases: proposition of a list based on the Brazilian Health System
Cássia Cunico & Silvana Nair Leite
Pages: 28-33
Published online: 16 Oct 2022
First Page PreviewforRare diseases: proposition of a list based on the Brazilian Health System | Full Text | References | PDF (204.2 KB) | EPUB
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Free Access

Review

Review
Targeting the IL-2 pathway for the treatment of mucosal melanoma
Ann C. Mercurio, Ashray B. Maniar, Alexander Z. Wei & Richard D. Carvajal
Pages: 34-47
Published online: 16 Oct 2022
AbstractforTargeting the IL-2 pathway for the treatment of mucosal melanoma | Full Text | References | PDF (4.4 MB) | EPUB
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Original Research

Article
Quality of life of patients with acromegaly: comparison of different therapeutic modalities
Yanitsa Rusenova, Silvia Vandeva, Atanaska Elenkova, Guenka Petrova & Maria Kamusheva
Pages: 48-54
Published online: 14 Dec 2022
AbstractforQuality of life of patients with acromegaly: comparison of different therapeutic modalities | Full Text | References | PDF (1.1 MB) | EPUB
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Systematic Review

Systematic Review
Making orphan drugs and services available and accessible for people who live with rare diseases: what has been done? a systematic scoping review
Hesam Ghiasvand, Maxwell S. Barnish, Tayebeh Moradi, Elham Nikram & Seyran Naghdi
Pages: 55-64
Published online: 14 Dec 2022
AbstractforMaking orphan drugs and services available and accessible for people who live with rare diseases: what has been done? a systematic scoping review | Full Text | References | PDF (306 KB) | EPUB | Supplemental
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Drug Evaluation

Article
Risdiplam as an orphan drug treatment of spinal muscular atrophy in adults and children (2 months or older)
N. Deconinck & E. Devos
Pages: 65-72
Published online: 23 Dec 2022
AbstractforRisdiplam as an orphan drug treatment of spinal muscular atrophy in adults and children (2 months or older) | Full Text | References | PDF (825.4 KB) | EPUB
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Open Access

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