Anne R. Pariser

Internal Medicine

2921 11th St S
Arlington, VA 22204

Last Updated: 09/26/2026

Save publications for later

Sign Up

Not sure about your diagnosis?

Check Your Symptoms

Found 33 publications

Non-oncologic orphan drug approvals across the world: Types of evidence required and time to approval.

Non-oncologic orphan drug approvals across the world: Types of evidence required and time to approval.

Journal: Drug discovery today

Published: August 14, 2025

The role of regulatory flexibility in the review and approval process of rare disease drug development.

The role of regulatory flexibility in the review and approval process of rare disease drug development.

Journal: Journal of biopharmaceutical statistics

Published: April 12, 2025

Use of alternative and confirmatory data in support of rare disease drug development.

Use of alternative and confirmatory data in support of rare disease drug development.

Journal: Journal of biopharmaceutical statistics

Published: April 12, 2025

Adeno-Associated Virus Gene Therapy Development: Early Planning and Regulatory Considerations to Advance the Platform Vector Gene Therapy Program.

Adeno-Associated Virus Gene Therapy Development: Early Planning and Regulatory Considerations to Advance the Platform Vector Gene Therapy Program.

Journal: Human gene therapy

Published: February 20, 2025

Increasing Diversity, Equity, Inclusion, and Accessibility in Rare Disease Clinical Trials.

Increasing Diversity, Equity, Inclusion, and Accessibility in Rare Disease Clinical Trials.

Journal: Pharmaceutical medicine

Published: June 14, 2024

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

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

Journal: Orphanet journal of rare diseases

Published: March 11, 2023

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.

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.

Journal: Human gene therapy

Published: January 25, 2023

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

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

Journal: Orphanet journal of rare diseases

Published: August 01, 2022

Global Regulatory and Public Health Initiatives to Advance Pediatric Drug Development for Rare Diseases.

Global Regulatory and Public Health Initiatives to Advance Pediatric Drug Development for Rare Diseases.

Journal: Therapeutic innovation & regulatory science

Published: February 18, 2022

Artificial Intelligence in Medical Imaging and its Impact on the Rare Disease Community: Threats, Challenges and Opportunities.

Artificial Intelligence in Medical Imaging and its Impact on the Rare Disease Community: Threats, Challenges and Opportunities.

Journal: PET clinics

Published: November 23, 2021

The national economic burden of rare disease in the United States in 2019.

The national economic burden of rare disease in the United States in 2019.

Journal: Orphanet journal of rare diseases

Published: November 03, 2021

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

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

Journal: Orphanet journal of rare diseases

Published: May 17, 2021

Showing 1-12 of 33

Last Updated: 09/26/2026