1.1(top 14%)
Impact Factor
1.2(top 14%)
extended IF
23(top 15%)
H-Index
607
authors
747
papers
2.6K
citations
1.1K
citing journals
3.4K
citing authors

Most Cited Articles of Expert Opinion on Orphan Drugs

TitleYearCitations
Orphan drugs and rare diseases: a scientometric review (2000 – 2014)2014105
PSEUDOXANTHOMA ELASTICUM: DIAGNOSTIC FEATURES, CLASSIFICATION, AND TREATMENT OPTIONS201449
Pathogenesis, Emerging therapeutic targets and Treatment in Sialidosis201541
Enzyme replacement therapy for treating mucopolysaccharidosis type IVA (Morquio A syndrome): effect and limitations201538
Pathogenesis, Epidemiology, Diagnosis and Clinical Aspects of Smith-Lemli-Opitz Syndrome201535
Diagnosis, prevalence, and screening of familial dilated cardiomyopathy201535
Clinical Features, Genetics and Potential Therapeutic Approaches for Birt-Hogg-Dubé Syndrome201535
Alveolar echinococcosis: evaluation of therapeutic strategies201432
Machado Joseph disease: clinical and genetic aspects, and current treatment201529
Gene Therapy for Duchenne muscular dystrophy201529
Emerging therapies and therapeutic concepts for lysosomal storage diseases201329
From mysteries to medicines: drug development for fibrodysplasia ossificans progressive201328
New therapeutic targets in rare genetic skeletal diseases201527
Hard targets for a second skeleton: therapeutic horizons for fibrodysplasia ossificans progressiva (FOP)201727
Pathogenic mechanisms and the prospect of gene therapy for choroideremia201526
The potential of utrophin modulators for the treatment of Duchenne muscular dystrophy201826
Tissue Chips to aid drug development and modeling for rare diseases201626
Genetics and prospective therapeutic targets for Sjögren-Larsson Syndrome201625
Therapies of mucopolysaccharidosis IVA (Morquio A syndrome)201324
Pharmacological therapeutics targeting the secondary defects and downstream pathology of Duchenne muscular dystrophy201624
Galactosialidosis: historic aspects and overview of investigated and emerging treatment options201723
Pathogenesis, diagnosis and therapeutic strategies in WHIM syndrome immunodeficiency201723
Obstacles and future of gene therapy for hemophilia201523
Gene therapy for the treatment of X-linked retinitis pigmentosa201823
Translating HDAC inhibitors in Friedreich's ataxia201623