Skip to main content
HHS Logo U.S. Department of Health & Human Services Divider arrow NIH logo National Institutes of Health Alt desc
Eric Wk Sid and Taj Neaz Powell participating on a panel.

Patient Views and Hope of Research Defined 10th NIH Rare Disease Day

On March 1, 2018, patients, scientists, policymakers and others participated in the 10th annual Rare Disease Day at NIH.

TRND Pompe image viewed on tablet

NCATS Collaboration Enables Gene Therapy to Move to Clinical Trial

NCATS scientists and NIH-funded researchers at Duke University collaborate to advance a gene therapy for Pompe disease into clinical testing for the first time.

Joe and Ella Murray

Rare Disease Patients and Families Find Hope in Research

NCATS supports rare disease patients and their communities by providing research funding, tools and other resources to help address their unique challenges.

Work with Us

TRND stimulates therapeutic development research collaborations among NIH and academic scientists, nonprofit organizations, and pharmaceutical and biotechnology companies working on rare and neglected illnesses. Learn more and contact TRND.

Therapeutics for Rare and Neglected Diseases (TRND)

The TRND program supports pre-clinical development of therapeutic candidates intended to treat rare or neglected disorders, with the goal of enabling an Investigational New Drug (IND) application. Learn more.

Find NCATS Programs & InitiativesAccess NCATS Expertise & Resources

About TRND

TRND supports pre-clinical development from lead optimization through IND application.

Scientific Capabilities

TRND offers world-class in-house experts and connections to external resources.

TRND Projects

Explore active and completed therapeutic development projects supported by TRND, including projects with clinical activities.

Why TRND Matters

There are more than 6,500 identified rare and neglected diseases, yet only about 250 treatments are available for these conditions. One reason is that limited numbers of patients can make gathering information and designing drug studies difficult. As a result, scientists often know little about the symptoms and biology of these conditions. Another obstacle is that some private companies may find it difficult to justify the cost of developing drugs for such small rare disease markets. Even in the case of infectious diseases — such as malaria — that inflict health burdens on large numbers of people in the developing world, the private sector often neglects therapeutic development because of insufficient economic incentives. Learn more about TRND.