The FDA creates a quicker path for gene therapies

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.

FDA proposes new treatment approval pathway for ultrarare diseases

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

My granddaughter has a rare disease. But clinical trials often exclude patients like her

Even when promising clinical trials exist, and the FDA allows expanded access, rare disease patients like my granddaughter ...

Study reveals why some immune disorders trigger severe food allergies, and others don't

A new study has shed light on why patients with certain rare immune disorders develop severe, food-triggered allergic reactions while others with similar diagnoses do not. The findings, published in ...

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

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

Don’t let hope expire for children with rare diseases

Across America, millions of parents of children with rare diseases are in a race against time, hoping that new treatments will be developed fast enough to save their kids. Thanks to advances in ...
CNBC

CNBC CURES DEBUTS WITH MISSION TO BUILD COMMUNITY AND RAISE AWARENESS FOR RARE DISEASES

Becky Quick leads CNBC Cures, bringing a deeply personal perspective to the multi-media initiative Weekly newsletter, videocast, and website share impactful stories and breaking news around the 30 ...
WDBJ

EARLY YEARS: Virginia teen with rare disease launches nonprofit to encourage youth to advocate for their heath

FAIRFAX COUNTY, Va. (WDBJ) - Classwork by day, A.I. innovation by night. Khartik Uppalapati is a young man with a mission. It all started when he was diagnosed with a rare lymphatic disorder in 2021 ...