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Therapeutics for Rare and Neglected Diseases (TRND)

Neena Nizar, seated in a wheelchair, speaks to the audience at Rare Disease Day at NIH 2020 about her experience with the rare disease metaphyseal chondrodysplasia.
Spreading a Message of Hope at Rare Disease Day at NIH

The annual event — held this year on Feb. 28, 2020 — focused on forging connections in the rare diseases community, shortening the journey toward diagnosis and pursuing personalized medicine as a therapeutic approach for treating rare diseases.

Novel Coronavirus SARS-CoV-2
OpenData Portal: Sharing Data to Advance COVID-19 Research

NCATS created a new resource for scientists, the OpenData Portal, to openly and quickly share COVID-19-related drug repurposing data and experiments for all approved drugs.

About TRND

TRND supports preclinical development from lead optimization through IND application.

Microscopic image showing dense pockets of lipid accumulations throughout a brain affected by Niemann-Pick Disease type c (NPC).
Scientific Capabilities

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

Microscopic image showing dense pockets of lipid accumulations throughout a brain affected by Niemann-Pick Disease type c (NPC).
TRND Projects

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

Photo of a scientist looking at vials of chemicals within a fume hood in a laboratory.
Microscopic image showing dense pockets of lipid accumulations throughout a brain affected by Niemann-Pick Disease type c (NPC).

About TRND

TRND supports preclinical development from lead optimization through IND application.

Microscopic image showing dense pockets of lipid accumulations throughout a brain affected by Niemann-Pick Disease type c (NPC).

Scientific Capabilities

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

Photo of a scientist looking at vials of chemicals within a fume hood in a laboratory.

TRND Projects

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

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.

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.

TRND Facts

Download the TRND fact sheet (PDF - 347KB).

Learn more about rare diseases research at NCATS.