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The need for worldwide policy and action plans for rare diseases

Acta Paediatrica, 2012-08, Vol.101 (8), p.805-807

2012

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Accelerating orphan drug development

Nature reviews. Drug discovery, 2010-12, Vol.9 (12), p.901-902

2010

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Tuberculosis following the Use of Etanercept, a Tumor Necrosis Factor Inhibitor

Clinical infectious diseases, 2004-08, Vol.39 (3), p.295-299

2004

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Primary melanomas of the esophagus and anorectum: epidemiologic comparison with melanoma of the skin

Melanoma research, 2009-02, Vol.19 (1), p.58-60

2009

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

Nature reviews. Drug discovery, 2010-07, Vol.9 (7), p.519-522

2010

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Database identifies FDA-approved drugs with potential to be repurposed for treatment of orphan diseases

Briefings in bioinformatics, 2011-07, Vol.12 (4), p.341-345

2011

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Regulatory considerations for developing drugs for rare diseases: orphan designations and early phase clinical trials

Discovery medicine, 2011-04, Vol.11 (59), p.367-375

2011

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Botulinum toxin type A injections: Adverse events reported to the US Food and Drug Administration in therapeutic and cosmetic cases

Journal of the American Academy of Dermatology, 2005-09, Vol.53 (3), p.407-415

2005

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Case reports of heart failure after therapy with a tumor necrosis factor antagonist

Annals of internal medicine, 2003-05, Vol.138 (10), p.807

2003

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Pathology and classification of ovarian tumors

Cancer, 2003-05, Vol.97 (S10), p.2631-2642

2003

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Infectious complications of biologic treatments of rheumatoid arthritis

Current opinion in rheumatology, 2003-05, Vol.15 (3), p.179-184

2003

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Primary malignant neoplasms of the appendix: A population-based study from the Surveillance, Epidemiology and End-Results Program, 1973-1998

Cancer, 2002-06, Vol.94 (12), p.3307-3312

2002

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Clinical pharmacology as a cornerstone of orphan drug development

Nature reviews. Drug discovery, 2011-11, Vol.10 (11), p.795-796

2011

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Autologous cultured chondrocytes: adverse events reported to the United States Food and Drug Administration

Journal of bone and joint surgery. American volume, 2006-03, Vol.88 (3), p.503

2006

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Spectrum of AIDS-associated malignant disorders

The Lancet (British edition), 1998-06, Vol.351 (9119), p.1833-1839

1998

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Duchenne muscular dystrophy: Drug development and regulatory considerations

Muscle & nerve, 2010-06, Vol.41 (6), p.740-745

2010

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Therapies for inborn errors of metabolism: what has the orphan drug act delivered?

Pediatrics (Evanston), 2010-07, Vol.126 (1), p.101-106

2010

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Endocrine tumors of the uterine cervix: incidence, demographics, and survival with comparison to squamous cell carcinoma

Gynecologic oncology, 2003-03, Vol.88 (3), p.333-339

2003

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The Orphan Drug Act and the Development of Stem Cell-Based Products for Rare Diseases

Cell stem cell, 2010-09, Vol.7 (3), p.283-287

2010

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Drug-induced systemic lupus erythematosus and TNF-α blockers

The Lancet (British edition), 2002, Vol.360 (9333), p.646-646

2002

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CK20 and CK7 protein expression in colorectal cancer: demonstration of the utility of a population-based tissue microarray

Human pathology, 2005-03, Vol.36 (3), p.275-281

2005

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Tissues from population-based cancer registries: a novel approach to increasing research potential

Human pathology, 2005-07, Vol.36 (7), p.812-820

2005

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Pure Red-Cell Aplasia and Recombinant Erythropoietin

The New England journal of medicine, 2002-05, Vol.346 (20), p.1584-1586

2002

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Survival among women with borderline ovarian tumors and ovarian carcinoma: A population-based analysis

Cancer, 2004-03, Vol.100 (5), p.1045-1052

2004

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Deaths among children less than two years of age receiving palivizumab: an analysis of comorbidities

The Pediatric infectious disease journal, 2004-04, Vol.23 (4), p.342-345

2004

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The need for worldwide policy and action plans for rare diseases

Accelerating orphan drug development

Tuberculosis following the Use of Etanercept, a Tumor Necrosis Factor Inhibitor

Primary melanomas of the esophagus and anorectum: epidemiologic comparison with melanoma of the skin

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

Database identifies FDA-approved drugs with potential to be repurposed for treatment of orphan diseases

Regulatory considerations for developing drugs for rare diseases: orphan designations and early phase clinical trials

Botulinum toxin type A injections: Adverse events reported to the US Food and Drug Administration in therapeutic and cosmetic cases

Case reports of heart failure after therapy with a tumor necrosis factor antagonist

Pathology and classification of ovarian tumors

Infectious complications of biologic treatments of rheumatoid arthritis

Primary malignant neoplasms of the appendix: A population-based study from the Surveillance, Epidemiology and End-Results Program, 1973-1998

Clinical pharmacology as a cornerstone of orphan drug development

Autologous cultured chondrocytes: adverse events reported to the United States Food and Drug Administration

Spectrum of AIDS-associated malignant disorders

Duchenne muscular dystrophy: Drug development and regulatory considerations

Therapies for inborn errors of metabolism: what has the orphan drug act delivered?

Endocrine tumors of the uterine cervix: incidence, demographics, and survival with comparison to squamous cell carcinoma

The Orphan Drug Act and the Development of Stem Cell-Based Products for Rare Diseases

Drug-induced systemic lupus erythematosus and TNF-α blockers

CK20 and CK7 protein expression in colorectal cancer: demonstration of the utility of a population-based tissue microarray

Tissues from population-based cancer registries: a novel approach to increasing research potential

Pure Red-Cell Aplasia and Recombinant Erythropoietin

Survival among women with borderline ovarian tumors and ovarian carcinoma: A population-based analysis

Deaths among children less than two years of age receiving palivizumab: an analysis of comorbidities

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