A incorporação de tecnologias em saúde para doenças raras no SUS
Jornal de Assistência Farmacêutica e FarmacoeconomiaIntrodução: Desde a vigência da Política Nacional de Doenças Raras, publicada pela Portaria GM/MS 199/2014 observa-se um crescente interesse do Ministério da Saúde do Brasil na estruturação de sua rede de cuidado no campo das doenças raras, bem como na ...
Tacila Pires Mega +1 more
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Drugs obtained by biotechnology processing [PDF]
, 2011 In recent years, the number of drugs of biotechnological origin available for many different diseases has increased exponentially, including different types of cancer, diabetes mellitus, infectious diseases (e.g.
Almeida, Hugo +2 more
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Diagnosing Hunter syndrome in pediatric practice: practical considerations and common pitfalls [PDF]
, 2012 Mucopolysaccharidosis II (MPS II), or Hunter syndrome, is an X-linked lysosomal storage disorder caused by a deficiency in the enzyme iduronate-2-sulfatase. Affected patients suffer progressive damage to multiple organ systems and early mortality.
A Keilmann +48 more
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Growth in expenditure on high cost drugs in Australia [PDF]
The paper provides an overview of the expenditure of high cost drugs in Australia and examines the average annual growth of these programs. The outlook for expenditure on high cost medicines and possible policy responses is also considered.
Rebecca de Boer, Roy Harvey
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Современные подходы к лечению синдрома Хантера [PDF]
, 2018 Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is an X-linked hereditary disorder associated with a deficiency of iduronate2-sulfatase (IDS). IDS deficiency provokes the accumulation of dermatan sulfate and heparan sulfate in different tissues ...
Alla N. Semyachkina +13 more
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Establishment of the effectiveness of early versus late stem cell gene therapy in Mucopolysaccharidosis II for treating central versus peripheral disease [PDF]
, 2023 Mucopolysaccharidosis type II (MPSII) is a rare paediatric X-linked lysosomal storage disease, caused by heterogeneous mutations in the IDS gene, which result in accumulation of heparan sulphate and dermatan sulphate within cells.
Bigger, Brian +5 more
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Impact of orphan drugs on Latvian budget [PDF]
, 2016 Publisher Copyright: © 2016 Logviss et al.Background: Number of orphan medicinal products on the market and number of rare disease patients, taking these usually expensive products, are increasing.
Krievins, Dainis +2 more
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Assessment of the impact of procurement centralization on the drug provision for patients within the High-Cost Nosology Program in the Russian Federation [PDF]
Background. In the Russian Federation, patients receive expensive drugs from the federal budget funds within High-Cost Nosology (HCN) Program.Objective: to assess the impact of drug procurement centralization on the weighted average price (WAP) per 1 ...
A. A. Kingshott +6 more
core +2 more sources
Relationship of Sleep to Pulmonary Function in Mucopolysaccharidosis II [PDF]
, 2013 To study the sleep characteristics, pulmonary function, and their relationships in an enzyme naive population of patients with mucopolysaccharidosis (MPS) II (Hunter syndrome)
Muenzer, Joseph +3 more
core +2 more sources
PRO40 BUDGET IMPACT ANALYSIS OF IDURSULFASE BETA IN THE TREATMENT MUCOPOLYSACCHARIDOSIS TYPE II IN THE RUSSIAN FEDERATION [PDF]
Value in Health, 2019 R. Yagudina +3 more
openaire +1 more source