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From scientific discovery to treatments for rare diseases - the view from the National Center for Advancing Translational Sciences - Office of Rare Diseases Research

Orphanet journal of rare diseases, 2018-11, Vol.13 (1), p.196-196, Article 196

2018

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The IDeaS initiative: pilot study to assess the impact of rare diseases on patients and healthcare systems

Orphanet journal of rare diseases, 2021-10, Vol.16 (1), p.429-429, Article 429

2021

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Operational description of rare diseases: a reference to improve the recognition and visibility of rare diseases

Orphanet journal of rare diseases, 2024-09, Vol.19 (1), p.334-6, Article 334

2024

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Towards the international interoperability of clinical research networks for rare diseases: recommendations from the IRDiRC Task Force

Orphanet journal of rare diseases, 2023-05, Vol.18 (1), p.109-109, Article 109

2023

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Cross-sector sponsorship of research in eosinophilic esophagitis: A collaborative model for rational drug development in rare diseases

Journal of allergy and clinical immunology, 2012-09, Vol.130 (3), p.613-616

2012

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Important Role of Translational Science in Rare Disease Innovation, Discovery, and Drug Development

Journal of general internal medicine : JGIM, 2014-08, Vol.29 (Suppl 3), p.804-807

2014

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Representation of Women and Minorities in Clinical Trials for New Molecular Entities and Original Therapeutic Biologics Approved by FDA CDER from 2013 to 2015

Journal of women's health (Larchmont, N.Y. 2002), 2018-04, Vol.27 (4), p.418-429

2018

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Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience

Human gene therapy, 2023-03, Vol.34 (5-6), p.217-227

2023

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Investigational New Drug applications: a 1-year pilot study on rates and reasons for clinical hold

Journal of investigative medicine, 2016-02, Vol.64 (2), p.376-382

2016

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A role for plasma cell targeting agents in immune tolerance induction in autoimmune disease and antibody responses to therapeutic proteins

Clinical immunology (Orlando, Fla.), 2016-04, Vol.165, p.55-59

2016

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Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction

Molecular genetics and metabolism, 2016-02, Vol.117 (2), p.66-83

2016

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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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Ketoconazole 2% shampoo in the treatment of tinea versicolor: A multicenter, randomized, double-blind, placebo-controlled trial

Journal of the American Academy of Dermatology, 1998-12, Vol.39 (6), p.944-950

1998

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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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The role of immune tolerance induction in restoration of the efficacy of ERT in Pompe disease

American journal of medical genetics. Part C, Seminars in medical genetics, 2012-02, Vol.160C (1), p.30-39

2012

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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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Clinical trial safety population size: analysis of drug approvals for rare and common indications by FDA Center for Drug Evaluation and Research

Expert opinion on orphan drugs, 2014-09, Vol.2 (9), p.869-875

2014

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A double-blind, placebo-controlled study of topical tetracaine in the treatment of herpes labialis

Journal of the American Academy of Dermatology, 1999-12, Vol.41 (6), p.996-1001

1999

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Characteristics of rare disease marketing applications associated with FDA product approvals 2006–2010

Drug discovery today, 2012-08, Vol.17 (15-16), p.898-904

2012

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A double-blind, randomized, placebo-controlled trial of adalimumab in the treatment of cutaneous sarcoidosis

Journal of the American Academy of Dermatology, 2013-05, Vol.68 (5), p.765-773

2013

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Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Lidocaine/Tetracaine Patch for Induction of Local Anesthesia prior to Minor Dermatologic Procedures in Geriatric Patients

Dermatologic surgery, 2005-03, Vol.31 (3), p.287-291

2005

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Safety of a new micronized formulation of isotretinoin in patients with severe recalcitrant nodular acne: A randomized trial comparing micronized isotretinoin with standard isotretinoin

Journal of the American Academy of Dermatology, 2001-08, Vol.45 (2), p.196-207

2001

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A randomized trial of the efficacy of a new micronized formulation versus a standard formulation of isotretinoin in patients with severe recalcitrant nodular acne

Journal of the American Academy of Dermatology, 2001-08, Vol.45 (2), p.187-195

2001

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Pre–Investigational New Drug Meetings With the FDA: Evaluation of Meeting Content and Characteristics of Applications for New Drug and Biologic Products

Therapeutic innovation & regulatory science, 2015-05, Vol.49 (3), p.434-442

2015

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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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From scientific discovery to treatments for rare diseases - the view from the National Center for Advancing Translational Sciences - Office of Rare Diseases Research

The IDeaS initiative: pilot study to assess the impact of rare diseases on patients and healthcare systems

Operational description of rare diseases: a reference to improve the recognition and visibility of rare diseases

Towards the international interoperability of clinical research networks for rare diseases: recommendations from the IRDiRC Task Force

Cross-sector sponsorship of research in eosinophilic esophagitis: A collaborative model for rational drug development in rare diseases

Important Role of Translational Science in Rare Disease Innovation, Discovery, and Drug Development

Representation of Women and Minorities in Clinical Trials for New Molecular Entities and Original Therapeutic Biologics Approved by FDA CDER from 2013 to 2015

Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience

Investigational New Drug applications: a 1-year pilot study on rates and reasons for clinical hold

A role for plasma cell targeting agents in immune tolerance induction in autoimmune disease and antibody responses to therapeutic proteins

Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction

Accelerating orphan drug development

Ketoconazole 2% shampoo in the treatment of tinea versicolor: A multicenter, randomized, double-blind, placebo-controlled trial

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

The role of immune tolerance induction in restoration of the efficacy of ERT in Pompe disease

Clinical pharmacology as a cornerstone of orphan drug development

Clinical trial safety population size: analysis of drug approvals for rare and common indications by FDA Center for Drug Evaluation and Research

A double-blind, placebo-controlled study of topical tetracaine in the treatment of herpes labialis

Characteristics of rare disease marketing applications associated with FDA product approvals 2006–2010

A double-blind, randomized, placebo-controlled trial of adalimumab in the treatment of cutaneous sarcoidosis

Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Lidocaine/Tetracaine Patch for Induction of Local Anesthesia prior to Minor Dermatologic Procedures in Geriatric Patients

Safety of a new micronized formulation of isotretinoin in patients with severe recalcitrant nodular acne: A randomized trial comparing micronized isotretinoin with standard isotretinoin

A randomized trial of the efficacy of a new micronized formulation versus a standard formulation of isotretinoin in patients with severe recalcitrant nodular acne

Pre–Investigational New Drug Meetings With the FDA: Evaluation of Meeting Content and Characteristics of Applications for New Drug and Biologic Products

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

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