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386,300 Results for "cure rare disease".
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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
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
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 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 pediatric disease priority review voucher program, among other initiatives, while ending a three-day partial government shutdown.
February 3, 2026
·
3 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
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
FDA
Calls for Leadership Stability and Rare Disease Follow-Through at FDA in 2026
After a tumultuous year, experts call for stability while anticipating the first fruits of policies intended to expedite approvals for rare disease drugs.
January 5, 2026
·
9 min read
·
Tristan Manalac
FDA
Rare Disease Leaders Call for Regulatory Consistency After Chaotic Year
As the FDA unveils a parade of initiatives aimed at accelerating drug development for rare diseases, experts appeal for a consistent approval process that will support and further catalyze momentum.
December 8, 2025
·
8 min read
·
Heather McKenzie
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
Press Releases
Cure Rare Disease Awarded $7.4 Million CIRM Grant to Advance Gene Therapy for Limb-Girdle Muscular Dystrophy Type 2i/R9
December 16, 2025
·
3 min read
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