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Literature DB >> 31312401

Novel Therapies for Orphan Diseases.

José M García Fernández¹, Carmen Ortiz Mellet².

Abstract

"Orphan" does not mean infrequent: over 7000 rare diseases affect millions of individuals. The US Orphan Drug Act and analogous regulations have succeeded at accelerating the development of novel therapies, but high prices threaten sustainability. Lysosomal storage disorders serve here to illustrate the light and shadows of this burgeoning field.

Entities: Disease

Year: 2019 PMID： 31312401 PMCID： PMC6627715 DOI： 10.1021/acsmedchemlett.9b00242

Source DB: PubMed Journal: ACS Med Chem Lett ISSN： 1948-5875 Impact factor: 4.345

10 in total

Review 1. The burden of rare diseases.

Authors: Carlos R Ferreira
Journal: Am J Med Genet A Date: 2019-03-18 Impact factor: 2.802

2. Drivers of Orphan Drug Development.

Authors: Ana Mingorance
Journal: ACS Med Chem Lett Date: 2018-10-01 Impact factor: 4.345

3. Outrageous prices of orphan drugs: a call for collaboration.

Authors: Lucio Luzzatto; Hanna I Hyry; Arrigo Schieppati; Enrico Costa; Steven Simoens; Franz Schaefer; Jonathan C P Roos; Giampaolo Merlini; Helena Kääriäinen; Silvio Garattini; Carla E Hollak; Giuseppe Remuzzi
Journal: Lancet Date: 2018-07-20 Impact factor: 79.321

Review 4. Emptying the stores: lysosomal diseases and therapeutic strategies.

Authors: Frances M Platt
Journal: Nat Rev Drug Discov Date: 2017-11-17 Impact factor: 84.694

Review 5. Chaperone therapy update: Fabry disease, GM1-gangliosidosis and Gaucher disease.

Authors: Yoshiyuki Suzuki
Journal: Brain Dev Date: 2013-01-03 Impact factor: 1.961

Review 6. Lysosomal storage disorders - challenges, concepts and avenues for therapy: beyond rare diseases.

Authors: André R A Marques; Paul Saftig
Journal: J Cell Sci Date: 2019-01-16 Impact factor: 5.285

Review 7. Enzyme replacement for lysosomal diseases.

Authors: Roscoe O Brady
Journal: Annu Rev Med Date: 2006 Impact factor: 13.739

8. Glycomimetic-based pharmacological chaperones for lysosomal storage disorders: lessons from Gaucher, GM1-gangliosidosis and Fabry diseases.

Authors: Elena M Sánchez-Fernández; José M García Fernández; Carmen Ortiz Mellet
Journal: Chem Commun (Camb) Date: 2016-04-25 Impact factor: 6.222

9. Estimating the clinical cost of drug development for orphan versus non-orphan drugs.

Authors: Kavisha Jayasundara; Aidan Hollis; Murray Krahn; Muhammad Mamdani; Jeffrey S Hoch; Paul Grootendorst
Journal: Orphanet J Rare Dis Date: 2019-01-10 Impact factor: 4.123

10. Investigating the landscape of US orphan product approvals.

Authors: Kathleen L Miller; Michael Lanthier
Journal: Orphanet J Rare Dis Date: 2018-10-22 Impact factor: 4.123