0.6(top 50%)
impact factor
19(top 100%)
papers
47(top 100%)
citations
4(top 100%)
h-index
0.6(top 50%)
impact factor
37
all documents
173
doc citations
7(top 100%)
g-index

Top Articles

#TitleJournalYearCitations
1Prussian blue as an antidote for radioactive thallium and cesium poisoningOrphan Drugs: Research and Reviews045
2Nitisinone: a reviewOrphan Drugs: Research and Reviews023
3Post-marketing access to orphan drugs: a critical analysis of health technology assessment and reimbursement decision-making considerationsOrphan Drugs: Research and Reviews201415
4A 30-year retrospective: National Organization for Rare Disorders, the Orphan Drug Act, and the role of rare disease patient advocacy groupsOrphan Drugs: Research and Reviews013
5Do ultra-orphan medicinal products warrant ultra-high prices? A reviewOrphan Drugs: Research and Reviews09
6Orphan drugs in glioblastoma multiforme: a reviewOrphan Drugs: Research and Reviews06
7Wilson’s disease: a review of treatment options with a focus on zinc therapyOrphan Drugs: Research and Reviews20125
8Stiripentol for the treatment of Dravet syndromeOrphan Drugs: Research and Reviews20145
9Drugs in development and dietary approach for Duchenne muscular dystrophyOrphan Drugs: Research and Reviews05
10Pompe disease: clinical perspectivesOrphan Drugs: Research and Reviews05
11Spotlight on anagrelide hydrochloride for the treatment of essential thrombocythemiaOrphan Drugs: Research and Reviews20175
12Current management options for tyrosinemiaOrphan Drugs: Research and Reviews04
13Access to cross-border health care services for patients with rare diseases in the European UnionOrphan Drugs: Research and Reviews20144
14Update on the challenges of treating trigeminal neuralgiaOrphan Drugs: Research and Reviews04
15Orphan drugs for sickle vaso-occlusion: dawn of a new era of targeted treatmentOrphan Drugs: Research and Reviews20154
16New treatments in development for Pseudomonas aeruginosa infections in the lungs of individuals with cystic fibrosisOrphan Drugs: Research and Reviews20143
17Betaine treatment of cystathionine β-synthase-deficient homocystinuria; does it work and can it be improved?Orphan Drugs: Research and Reviews20122
18Optimal management of interstitial lung disease associated with dermatomyositis/polymyositis: lessons from the Japanese experienceOrphan Drugs: Research and Reviews02
19Orphan drugs in development for urea cycle disorders: current perspectivesOrphan Drugs: Research and Reviews02
20Angiogenesis inhibitors in gastric cancerOrphan Drugs: Research and Reviews20142
21Fahr's disease: current perspectivesOrphan Drugs: Research and Reviews20152
22Adverse events reported for hereditary angioedema medications: a retrospective study of spontaneous reports submitted to the EudraVigilance database, 2007-2013Orphan Drugs: Research and Reviews02
23An update on gene therapy for the treatment of lipoprotein lipase deficiencyOrphan Drugs: Research and Reviews01
24Profile of aminopyridines for Lambert–Eaton myasthenic syndromeOrphan Drugs: Research and Reviews01
25Amyotrophic lateral sclerosis: clinical perspectivesOrphan Drugs: Research and Reviews20151
26Paroxysmal nocturnal hemoglobinuria: new concepts in pathophysiology and treatmentOrphan Drugs: Research and Reviews20151
27Orphan drugs for the treatment of aspergillosis: focus on isavuconazoleOrphan Drugs: Research and Reviews20171
28Medical treatment of rare diseases must be put on the agendaOrphan Drugs: Research and Reviews20110
29Potential role of enzastaurin in the treatment of patients with relapsed or refractory advanced cutaneous T-cell lymphomas: a reviewOrphan Drugs: Research and Reviews20120
30Tipifarnib and farnesyltransferase inhibitors in the treatment of inflammatory breast cancer: is the story over? A reviewOrphan Drugs: Research and Reviews00
31Orphan drugs in development for primary biliary cirrhosis: challenges and progressOrphan Drugs: Research and Reviews20150
32The efficacy and safety of adalimumab in ocular inflammatory diseaseOrphan Drugs: Research and Reviews20150
33Denosumab in the treatment of hypercalcemia secondary to malignancyOrphan Drugs: Research and Reviews20150
34Orphan drugs in development for long-chain fatty acid oxidation disorders: challenges and progressOrphan Drugs: Research and Reviews00
35Profile of lumacaftor/ivacaftor combination: potential in the treatment of cystic fibrosisOrphan Drugs: Research and Reviews00
36Orphan drugs in development for Huntington's disease: challenges and progressOrphan Drugs: Research and Reviews20150