Novel treatments for rare rheumatologic disorders: analysis of the impact of 30 years of the US orphan drug act

Novel Treatments for Rare Cancers: The U.S. Orphan Drug Act Is Delivering—A Cross-Sectional Analysis

(2016) Clemens Stockklausner et al.   ONCOLOGIST

Instruments for assessment of impairments and activity limitations in patients with hand conditions: A European Delphi study

(2015) L Ven-Stevens et al.   JOURNAL OF REHABILITATION MEDICINE

Pressure for drug development in lysosomal storage disorders – a quantitative analysis thirty years beyond the US orphan drug act

(2015) Konstantin Mechler et al.   Orphanet Journal of Rare Diseases

Biosimilars in Oncology: From Development to Clinical Practice

(2014) Katherine H. Rak Tkaczuk et al.   SEMINARS IN ONCOLOGY

Prevalence and incidence of juvenile idiopathic arthritis: A systematic review

(2013) Sigrid Thierry et al.   JOINT BONE SPINE

Characteristics of orphan drug applications that fail to achieve marketing approval in the USA

(2010) Harald E. Heemstra et al.   DRUG DISCOVERY TODAY

Translation of rare disease research into orphan drug development: disease matters

(2009) Harald E. Heemstra et al.   DRUG DISCOVERY TODAY

Is there a future for small molecule drugs in the treatment of rheumatic diseases?

(2008) Joanna Stanczyk et al.   CURRENT OPINION IN RHEUMATOLOGY

Does orphan drug legislation really answer the needs of patients?

(2008) Marlene E Haffner et al.   LANCET

Clinical trials of orphan medicines

(2008) Brendan M Buckley   LANCET

Why rare diseases are an important medical and social issue

(2008) Arrigo Schieppati et al.   LANCET