'Shaped my family’s life': Your rare disease stories

Our inbox has been flooded with emails from viewers and readers who told us how their lives have been affected by rare ...
Fierce Pharma

FDA’s rare pediatric disease voucher program revived by 2026 government funding bill

The Consolidated Appropriations Act of 2026, signed by Trump on Feb. 3, includes a provision reinstating the Mikaela Naylon ...
BioSpace

Rare Pediatric Disease Vouchers Reauthorized, PBM Reform Funded in Narrowly Passed Spending Bill

U.S. President Donald Trump signed a spending package into law Tuesday that reauthorizes the FDA’s previously stalled rare ...

TrumpRx must work for families with rare diseases | Opinion

The author's son has hemophilia, a rare disorder requiring expensive, life-saving medication. The TrumpRx program aims to ...

What is legionnaires’ disease? Norwegian Encore passengers diagnosed with rare illness after cruising last year

Two passengers contracted Legionnaires disease following a voyage on the Norwegian Encore in December 2025. Norwegian Cruise ...
Mammoth Times

5 Rare Kidney Diseases You May Not Know About

This Rare Disease Day, observed on Feb. 28 worldwide, the American Kidney Fund is committed to improving the understanding of ...

Machesney Park woman pleads for better care after rare disorder leaves her blind

The Machesney Park resident estimates spending nearly $1000 per month on eye treatments to avoid irritation; one regimen ...

US FDA declines to approve Regenxbio's gene therapy for rare disease

Feb 9 (Reuters) - Regenxbio said on Monday the U.S. Food and Drug Administration had declined to approve its gene therapy for ...
Morningstar

National Organization for Rare Disorders Adds Seven Rare Disease Centers of Excellence Nationwide

NORD's growing network, now 46 institutions strong, represents a coordinated nationwide effort to strengthen rare disease care and research collaboration With these new members, the network now ...

How AI is helping solve the labor issue in treating rare diseases

At Web Summit Qatar, AI-powered biotech startups describe how automation, data, and gene editing are filling labor gaps in ...
BioSpace

From Awareness to Acceleration: Rare Disease Drug Development Enters a Pivotal Era

Investor enthusiasm and evolving FDA pathways are accelerating rare disease drug development, with ultrarare conditions like ...