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Open access

Research article

First published 2023-03

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

Richa Madan Lomash, Oleg Shchelochkov, Randy J. Chandler [...]

Human Gene Therapy

Retricted access

Review article

First published 2022-3

US Food and Drug Administration Facilitated Pediatric Approval Programs: Application to Pediatric Neurological Disorders

Emily Da Cruz, Faraan Rahim, Monica Lemmon [...]

Journal of Child Neurology

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Review article

First published 2021-6

Challenges in Evaluating Safety and Efficacy in Drug Development for Rare Diseases: A Review for Pharmacists

Kanya K. Shah, Stephen Kogut, Angela Slitt

Journal of Pharmacy Practice

Open access

Research article

First published 2017-3

Characteristics of pediatric pulmonary hypertension trials registered on ClinicalTrials.gov

Jordan D. Awerbach, Richard A. Krasuski, Kevin D. Hill

Pulmonary Circulation

Open access

Research article

First published 2021

Family Perspectives on Clinical Research for Pediatric Multiple Sclerosis: Enhancing Equity

Leslie A Mandel, Ellen O’Donnell, Katia Canenguez [...]

Journal of Patient Experience

Retricted access

Review article

First published 2020-7

Tackling rare diseases: Clinical trials on chips

Sandra H Blumenrath, Bo Y Lee, Lucie Low [...]

Experimental Biology and Medicine

Open access

Review article

First published 2020

Precision medicine and its implementation in patients with NTRK fusion genes: perspective from developing countries

Andrés F. Cardona, Oscar Arrieta, Alejandro Ruiz-Patiño [...]

Therapeutic Advances in Respiratory Disease

Retricted access

Brief report

First published 2011-08

Antipsychotic Drug Administration Does Not Correlate with Prolonged Rate-Corrected QT Interval in Children and Adolescents: Results from a Nested Case–Control Study

Christoph U. Correll, Jennifer Harris, Vicki Figen [...]

Journal of Child and Adolescent Psychopharmacology

Free access

Research article

First published 2024-2

Utility of a Diabetes Medication Poster in a Primary Care Clinic

Wasif E. Shamsi, Tracey L. Mersfelder, Kevin J. Kavanaugh

Journal of Pharmacy Practice

Retricted access

Review article

First published 2024-4

Atezolizumab as the First Systemic Therapy Approved for Alveolar Soft Part Sarcoma

Emilie J. Bergsma, Mariam Elgawly, David Mancuso [...]

Annals of Pharmacotherapy

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