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674,907 Results for "rare disease therapeutics inc".
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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
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
FDA
Busy FDA gives rare disease sector complementary pathways, unanswered questions
The FDA has expanded the regulatory toolkit during President Donald Trump’s second term, adding new mechanisms for rare diseases while putting the Biden-era platform technology designation into action.
June 8, 2026
·
8 min read
·
Nick Paul Taylor
FDA
Post-Makary, new rare disease framework faces fresh uncertainty
As the FDA tries to clarify its intent for former FDA Commissioner Marty Makary’s plausible mechanism framework for bespoke therapies, experts emphasize the importance of expanding its scope to encompass rare diseases that affect more than just one or a few individuals.
June 8, 2026
·
7 min read
·
Heather McKenzie
Press Releases
Monopar Therapeutics Receives FDA Rare Pediatric Disease Designation for ALXN1840 for the Treatment of Wilson Disease
June 30, 2026
·
5 min read
Press Releases
Cadrenal Therapeutics to File for FDA Rare Pediatric Disease Designation for Tecarfarin in Kawasaki Disease
June 18, 2026
·
8 min read
Press Releases
Ipsen completes acquisition of Memo Therapeutics AG, adding first-in-class asset to Ipsen’s Rare Disease pipeline
July 22, 2026
·
7 min read
Opinion
Balancing Safety Access in Rare Disease—Lessons From Sarepta
Regulators overseeing rare disease treatments need better tools to weigh competing risks in real time. Sarepta Therapeutics’ Elevidys is a prime example of why.
February 25, 2026
·
6 min read
·
Joshua R. Mansbach
Regulatory
FDA delays verdict on Beren’s rare disease drug by three months
Beren Therapeutics is seeking approval in Neimann-Pick disease type C, an application the FDA was set to decide on by August 17. The new target action date is November 17.
May 29, 2026
·
2 min read
·
Tristan Manalac
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