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Review 1. Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years.

Authors: M Miles Braun; Sheiren Farag-El-Massah; Kui Xu; Timothy R Coté
Journal: Nat Rev Drug Discov Date: 2010-06-07 Impact factor: 84.694

Review 2. The importance of new companies for drug discovery: origins of a decade of new drugs.

Authors: Robert Kneller
Journal: Nat Rev Drug Discov Date: 2010-10-29 Impact factor: 84.694

Review 3. Rare diseases, orphan drugs and their regulation: questions and misconceptions.

Authors: Erik Tambuyzer
Journal: Nat Rev Drug Discov Date: 2010-11-09 Impact factor: 84.694

4. Policy alternatives for treatments for rare diseases.

Authors: Abbas H Panju; Chaim M Bell
Journal: CMAJ Date: 2010-07-12 Impact factor: 8.262

5. Availability of and access to orphan drugs: an international comparison of pharmaceutical treatments for pulmonary arterial hypertension, Fabry disease, hereditary angioedema and chronic myeloid leukaemia.

Authors: Carl Rudolf Blankart; Tom Stargardt; Jonas Schreyögg
Journal: Pharmacoeconomics Date: 2011-01 Impact factor: 4.981

6. Effect of Mexiletine on Muscle Stiffness in Patients With Nondystrophic Myotonia Evaluated Using Aggregated N-of-1 Trials.

Authors: Bas C Stunnenberg; Joost Raaphorst; Hans M Groenewoud; Jeffrey M Statland; Robert C Griggs; Willem Woertman; Dick F Stegeman; Janneke Timmermans; Jaya Trivedi; Emma Matthews; Christiaan G J Saris; Bas J Schouwenberg; Gea Drost; Baziel G M van Engelen; Gert Jan van der Wilt
Journal: JAMA Date: 2018-12-11 Impact factor: 56.272

Review 10. Garrod's Croonian Lectures (1908) and the charter 'Inborn Errors of Metabolism': albinism, alkaptonuria, cystinuria, and pentosuria at age 100 in 2008.

Authors: Charles R Scriver
Journal: J Inherit Metab Dis Date: 2008-10-12 Impact factor: 4.982

Adopting orphan drugs--two dozen years of treating rare diseases.