The Food and Drug Administration aims to evaluate treatments for rare diseases based on plausible evidence that they would work — without requiring a clinical trial first.

Federal health officials on Monday laid out a proposal to spur development of customized treatments for patients with hard-to-treat diseases, including for rare genetic conditions that the ...

The Food and Drug Administration (FDA) announced a new proposal Monday for flexible drug approval pathway treatments ...

St. Louis Families Unite for Rare Disease Day 2026 as Take Part Foundation Advances Critical Genetic Testing and ...

In many countries, newborn screening programmes test babies for certain inherited conditions shortly after birth.

Researchers have found that a new base-editing gene therapy can help treat a rare neurodevelopmental disorder called Snijders Blok–Campeau syndrome caused by mutations in the CHD3 gene. A specialized ...

I thought the hardest part of this journey would be to find a cure and a treatment for my son,” Andrew Jedlicka told The Post ...

Engineers at the University of Pennsylvania and Rice University have refined a technology for editing individual genetic ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...

For more than six decades, biomarker-based newborn screening has played a pivotal role in reducing infant mortality and ...

Genetic testing can help doctors diagnose some neurological disorders, such as Huntington’s disease. It can also provide insights into a person’s risk of future health conditions. Genetic testing ...