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650,715 Results for "national organization for rare disorders".
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Press Releases
National Organization for Rare Disorders Ranks All 50 States on Rare Disease Policies
February 10, 2026
·
4 min read
Press Releases
National Organization for Rare Disorders Adds Seven Rare Disease Centers of Excellence Nationwide
December 4, 2025
·
3 min read
Press Releases
National Organization for Rare Disorders Announces New Board Members
July 23, 2025
·
3 min read
Deals
Moderna Teams With Recordati on Rare Disease mRNA Therapy for up to $160M
Moderna will continue to lead clinical development and manufacturing of the asset, while Recordati will handle commercialization of mRNA-3927, which is under development for the rare metabolic disorder propionic acidemia.
January 30, 2026
·
1 min read
·
Annalee Armstrong
Drug Development
Biopharma Momentum To Propel Rare Disease, Cancer, Neuro Forward in 2026
After years stuck in the “doldrums,” the biopharma sector is in a “very good place” heading into the new year, analysts told
BioSpace
, with both rare and chronic diseases headlining investor and R&D interest as JPM26 kicks off.
January 12, 2026
·
6 min read
·
Heather McKenzie
Press Releases
National Institutes of Health Awards $1.54M Grant To Scottish Rite for Children for Rare Diseases Research
January 8, 2026
·
3 min read
Rare diseases
JPM Day 3: Rare Disease in the Spotlight
2026 is shaping up to be a pivotal year for rare disease drugmakers, with key approvals, filings and readouts lined up for this year.
January 15, 2026
·
5 min read
·
Tristan Manalac
Insights
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 MPS II moving into the spotlight.
February 4, 2026
·
3 min read
·
Jennifer Smith-Parker
Rare diseases
Rare Disease Biotechs Stand To Lose $4B if Priority Voucher Program Not Reinstated: Report
Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis by the Rare Disease Company Coalition shows. That could mean $4 billion in missed revenue for already cash-strapped biotechs.
January 26, 2026
·
7 min read
·
Heather McKenzie
Rare diseases
Rare Disease Vouchers Caught in Political Abyss. It Didn’t Have To Be This Way
The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates have been fighting to get it back ever since.
January 23, 2026
·
3 min read
·
Heather McKenzie
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