FDA Office of the Commissioner seal with US Food and Drug Administration text

REGULATORY

7 May 2026

One and Done: FDA Rips Up the Two-Trial Playbook

The FDA now accepts a single pivotal trial as its default for drug approval, a shift that could transform rare disease development

Chiesi pharmaceutical company logo on exterior building signage

PARTNERSHIPS

5 May 2026

The Oral Revolution for Rare Disease Is Here

Chiesi acquires KalVista for $1.9 billion, securing the first oral on-demand treatment for hereditary angioedema and sidelining painful in...

Corcept Therapeutics laboratory equipment used in advanced biomedical research

INNOVATION

1 May 2026

The Gene Fix That's Rewriting a Death Sentence for Kids

FDA approves Kresladi, a one-time gene therapy for LAD-I, a rare immune disorder that once left children with few options and little hope

GENA by Sivotec exhibition stand with three delegates meeting

TECHNOLOGY

29 Apr 2026

Ten Seconds to an Answer Years in the Making

A Florida startup's AI platform cuts rare genetic disease diagnosis from days to seconds and it's already been used in 162,000 cases acros...

Regeneron Pharmaceuticals corporate office building exterior

REGULATORY

23 Apr 2026

Breaking the Quiet With a Landmark Genetic Milestone

FDA approves Otarmeni, a groundbreaking gene therapy from Regeneron that restores natural hearing to children with rare genetic mutations

Lab technician in blue glove inserting a sample vial into branded clinical storage trays

PARTNERSHIPS

21 Apr 2026

Curing the Rare Disease Data Crisis

Lunai Bioworks and Geneial join forces to transform fragmented patient records into structured assets, accelerating cures for rare disorde...

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