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Rare Disease · news · pharmadog
← back to Rare Disease
News mentioning Rare Disease
345
stories
Sep 3, 26
Opinion: STAT+: A surprising bright spot at the FDA on regulating unproven stem cells
stat
Sep 3, 26
STAT+: FDA pilot offers generative AI medical devices a path to patients before they are authorized
stat
Sep 3, 26
Capricor, Replimune illustrate why FDA must listen to rare disease specialists
biospace
Sep 2, 26
STAT+: Ultragenyx drug to treat Angelman syndrome, a rare disease, fails late-stage trial
stat
Sep 2, 26
FDA raises bar for foreign site inspections as China trials surge
biospace
Sep 2, 26
Fortrea boosts early-phase CRO platform with $45M acquisition of Worldwide division
fiercebiotech
Sep 2, 26
UniQure files for FDA, UK approval of first gene therapy for Huntington’s disease
biospace
Sep 2, 26
Tavneos no longer available for new patients in UK as Amgen awaits FDA hearing
biospace
Sep 2, 26
Novartis’ and BMS’ paused CAR T trials, RevMed’s pancreatic cancer approval, more MFN deals
biospace
Sep 1, 26
GSK to launch Phase 3 trial for mRNA-based flu vaccine amid resurgence for the modality
biospace
Sep 1, 26
As MAHA champions nutrition, it’s ignoring the experts, dietitians say
stat
Aug 31, 26
ESC ‘26: Cytokinetics’ sell-off, Arrowhead’s edge and a heart drug autopsy
biopharma dive
Aug 31, 26
STAT+: We’re reading about drug pricing deals, a new cancer therapy approval, and more
stat
Aug 31, 26
BMS’ neuro renaissance was built on back of Celgene mega-merger
biospace
Aug 28, 26
BioNTech halts mid-stage trial of personalized mRNA vaccine in colorectal cancer
biospace
Aug 28, 26
Lilly joins Novo in cardiovascular space after FDA nod for GLP-1/GIP agonist Mounjaro
biospace
Aug 27, 26
STAT+: FDA approves Roivant therapy for rare autoimmune disease
stat
Aug 27, 26
Chutes & Ladders—Vivace revs up C-suite ahead of pivotal push
fiercebiotech
Aug 27, 26
STAT+: The mystery of Moderna’s magic algorithm
stat
Aug 26, 26
Biohaven trades cash for ‘upside’ with $795M epilepsy drug deal
biopharma dive
Aug 26, 26
Biohaven could get up to $795M in epilepsy alliance with SK Bio
biospace
Aug 25, 26
STAT+: Turkish antitrust authorities probe Teva for allegedly gaming the patent system, disparaging rival drugs
stat
Aug 25, 26
STAT+: Pharmalittle: We’re reading about RFK Jr. vaccine disclosures, a Merck loss over Medicare prices, and more
stat
Aug 24, 26
REGENXBIO’s Hunter syndrome gene therapy hit by FDA hold, stymieing resubmission plans
biospace
Aug 24, 26
STAT+: Pharmalittle: We’re reading about Medicaid obesity-drug coverage, a Regenxbio gene therapy, and more
stat
Aug 24, 26
STAT+: FDA pauses Regenxbio gene therapy trial again due to safety concerns
stat
Aug 24, 26
Spruce’s successful FDA talks plant seeds for Q4 filing of ultra-rare disease drug
fiercebiotech
Aug 24, 26
STAT+: Inside Moderna and Merck’s cancer vaccine triumph
stat
Aug 24, 26
Regenxbio gene therapy refiling plan derailed by FDA hold over spine MRI findings
fiercebiotech
Aug 24, 26
Capricor, Replimune reviews signal FDA’s return to reliability but every application will stand alone
biospace
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