CG Life and 24/364 officially launched the inaugural 24/364 Rare Disease Film Festival (RDFF), a first-of-its-kind cultural ...

Mast and Herper have covered rare disease issues for a combined three decades. As part of the reporting, Mast spent two days in the Bay Area with Matt Wilsey and Grace. Jason is a general assignment ...

Chan Zuckerberg Initiative's Biohub will offer new grants to the rare disease community while expanding its AI drug repurposing partnership with Every Cure.

With a regulatory route now in place, Regenxbio expects to meet with the FDA again in July, with a BLA resubmission to follow in Q3 2026.

The seven-year medical odyssey that once defined rare disease diagnosis is being rewritten by algorithms capable of identifying medical zebras in the time it takes to refresh your social media feed.

The federal agency ARPA-H earmarked $43 million for Philadelphia researchers to advance gene therapies for rare diseases.

In Sweden, more than 500,000 individuals live with a rare condition. Globally, approximately 7,000 distinct rare diseases ...

A Colorado family is sharing the story of their 5-year-old son's sudden death during a vacation, hoping greater awareness of ...

Over a thousand children visit Boston Children’s Hospital every day. Many get clear diagnoses and begin treatment, but a small subset of pediatric visitors with rare illnesses never get diagnoses at ...

Actor Colin Farrell is launching a new foundation to raise awareness of a rare genetic condition called Angelman syndrome, so that his son and others with the disorder will have more support and ...

The FDA determined the condition qualifies as a rare pediatric disease because it is a serious disorder that primarily affects patients from birth through age 18, Vanda said. The ...

A first-of-its-kind drug may slow the progression of Huntington's disease, a rare, fatal, neurodegenerative disorder.