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679,354 Results for "rare disease therapeutics inc".
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Rare diseases
Mikhail, Davis on board with regulatory framework tailored to rare disease therapies
Newly permanent CBER and CDER directors Karim Mikhail and Michael Davis are widely expected to continue the FDA’s efforts toward an adaptive framework for rare disease therapies. If approved by the Senate, will commissioner nominee Heidi Overton stay the course?
September 21, 2026
·
6 min read
·
Heather McKenzie
Press Releases
Secretome Therapeutics Expands Rare Disease Leadership Team
July 27, 2026
·
4 min read
FDA
FDA’s uniQure, REGENXBIO reversals could bolster other near-term rare disease applications
The FDA’s recently altered outlook on the evidence required for approval of rare disease drugs could have immediate benefits for companies including Skyhawk Therapeutics, Capricor Therapeutics and Biohaven.
June 25, 2026
·
6 min read
·
Heather McKenzie
Press Releases
ReviR Therapeutics Receives FDA Rare Pediatric Disease Designation for RTX-117 in Charcot-Marie-Tooth Disease
August 26, 2026
·
4 min read
Press Releases
Revive Therapeutics to Expand Bucillamine IP and Orphan Drug Portfolio Across Medical Countermeasures and Rare Diseases
September 15, 2026
·
6 min read
China
US ‘eroding’ lead in rare diseases a matter of national security: Report
A Congressional advisory panel recommends the U.S. government put supportive policies in place for rare disease drug development, such as a finalized platform designation from the FDA and the allowance of novel trial designs for small patient populations.
July 22, 2026
·
4 min read
·
Tristan Manalac
Rare diseases
Through flux and fluidity, FDA keeps coming back to rare disease
Even as FDA approvals for biologic therapies fell in the first half of 2026, regulatory experts are optimistic about a turnaround in the rare disease space after the departure of key leaders at the agency. Still, there will continue to be tension between science and politics.
July 8, 2026
·
6 min read
·
Heather McKenzie
Press Releases
NORD and Rosenau Family Research Foundation Launch Rare Research Launchpad to Introduce Students to Careers in Rare Disease Research
September 15, 2026
·
5 min read
Opinion
Capricor, Replimune illustrate why FDA must listen to rare disease specialists
Last month’s FDA advisory committee meetings for therapies developed by Capricor Therapeutics and Replimune effectively illustrate the different outcomes for patients when the agency prioritizes specialization in its reviews.
September 3, 2026
·
6 min read
·
Shahriar Minokadeh
Press Releases
Akebia Therapeutics Announces First Patient Dosed in Phase 2 Basket Trial of Ebribafusp in Rare Complement-Mediated Kidney Diseases
September 14, 2026
·
7 min read
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