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Rare Disease · news · pharmadog
← back to Rare Disease
News · Rare Disease
846
stories
Sep 3, 26
STAT+: Ionis wins FDA approval for first drug for Alexander disease, a rare and deadly neurological disorder
stat
Sep 3, 26
FDA Approves First Drug to Treat Alexander Disease
fda press
Sep 3, 26
An AI drug specialist zeroes in on rare forms of obesity
biopharma dive
Sep 3, 26
Chutes & Ladders—Invivyd's chairman adds CEO role ahead of COVID readout
fiercebiotech
Sep 3, 26
Ultragenyx to weigh ‘significant’ cost cuts as Angelman drug fails key study
biopharma dive
Sep 3, 26
STAT+: Seattle scientists launch $95 million AI biology effort
stat
Sep 3, 26
STAT+: Fauci speaks, and writes
stat
Sep 3, 26
RevMed’s newly approved Rasonque could ‘hold up’ in non-small cell lung cancer
biospace
Sep 3, 26
US to grant up to $125M for personalized RNA manufacturing innovation
biospace
Sep 3, 26
STAT+: We’re reading about an Ultragenyx failure, RevMed for lung cancer, and more
stat
Sep 3, 26
Ultragenyx’s 'high-risk' Angelman bet fails, cratering stock and forcing strategic review
biospace
Sep 3, 26
Replimune’s melanoma drug would have been rejected again if not for senior CBER official
biospace
Sep 3, 26
AbbVie’s bispecific T-cell engager shows promise in ph. 3 multiple myeloma trial
fiercebiotech
Sep 3, 26
The pediatricians who take in vaccine-skeptical parents others won’t
stat
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 3, 26
Ultragenyx fails phase 3 Angelman test, plots ‘significant expense reductions’
fiercebiotech
Sep 3, 26
Ultragenyx’s Angelman therapy fails Phase 3 trial in setback for company and patients
endpoints
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 2, 26
Convenience Kit Correction: Medline Issues Correction for Convenience Kits Containing BD ChloraPrep Applicators
fda medwatch
Sep 1, 26
STAT+: House votes to temporarily block Trump plan to politicize federal grants
stat
Sep 1, 26
GSK to launch Phase 3 trial for mRNA-based flu vaccine amid resurgence for the modality
biospace
Sep 1, 26
STAT+: Will House delay Trump plan to politicize NIH grants?
stat
Sep 1, 26
9 midsize drugmakers climb aboard Trump’s MFN train
biospace
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