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Rare Disease · news · pharmadog
← back to Rare Disease
News · Rare Disease
855
stories
Jul 28, 26
Incyte ditches phase 1-stage JAK inhibitor to pursue next-gen options
fiercebiotech
Jul 28, 26
STAT+: Preliminary trial results suggest a path to a safer Alzheimer’s treatment
stat
Jul 28, 26
STAT+: From ‘lost cause’ to gold rush: Biotechs swarm to cure AATD
stat
Jul 28, 26
As pharma descends on China for deals, BIOSECURE Act raises intellectual property risk
biospace
Jul 27, 26
STAT+: Pharma’s drug spending deflection
stat
Jul 27, 26
‘This could be a large market’: Novo Holdings-backed Claris secures $118M for corneal disease drug
fiercebiotech
Jul 27, 26
UPDATE: FDA disputes Capricor's claims of Phase 3 success for DMD cell therapy
biospace
Jul 27, 26
Gossamer regains rights to lung disease asset from Chiesi after Phase 3 fail
biospace
Jul 27, 26
FDA scientists question Capricor data; Legend CEO steps down
biopharma dive
Jul 27, 26
Sarepta names former AbbVie R&D exec Michael Severino as next CEO
biospace
Jul 27, 26
RA Capital launches rare disease company Oak Hill Bio onto Nasdaq via SPAC
fiercebiotech
Jul 27, 26
STAT+: Sarepta names Michael Severino, former Tessera CEO, as new chief executive
stat
Jul 27, 26
STAT+: Pharmalittle: We’re reading about a U.S. peptide panel’s votes, Amgen scuffling with the FDA, and more
stat
Jul 27, 26
STAT+: Ahead of hearing, FDA questions the efficacy of Capricor’s Duchenne drug
stat
Jul 27, 26
AstraZeneca has no plans to ramp up dealmaking in wake of Wainua failure
biospace
Jul 27, 26
A trend that isn’t fading, ultra-processed baby foods
stat
Jul 27, 26
STAT+: RA Capital backs upstart rival to Ultragenyx and Ionis in rare brain disease
stat
Jul 27, 26
Updated: AstraZeneca foresees blockbuster sales for Claudin 18.2 ADC, but Ultomiris flops in rare disease
endpoints
Jul 27, 26
After missed endpoint, Gossamer and Chiesi pull the plug on PAH drug collaboration
fiercebiotech
Jul 27, 26
Opinion: How midwives like me can help fight medical misinformation
stat
Jul 27, 26
STAT+: As some medical schools embrace RFK Jr.’s nutrition push, they deftly sidestep MAHA priorities
stat
Jul 27, 26
Summit, Merck must navigate new regulatory terrain in China-first cancer landscape
biospace
Jul 24, 26
FDA advisory panel narrowly rejects compounding of one peptide, backs two others
stat
Jul 24, 26
Amgen shares new Tavneos analysis in effort to resolve FDA dispute
endpoints
Jul 24, 26
Amgen sheds around 40 staffers amid continuing struggles
fiercebiotech
Jul 24, 26
Ipsen’s Bylvay fails key trial in children with rare liver disease
endpoints
Jul 24, 26
Sanofi’s $1.4B Kymab bet falters again as atopic dermatitis trials scrapped
biospace
Jul 24, 26
Scribe reels in nearly $129M in rare early-stage IPO for a gene editing biotech
biospace
Jul 24, 26
Amgen deploys patients to defend troubled Tavneos in FDA hearing request
biospace
Jul 24, 26
STAT+: Fatal gene-editing trial in China raises ethics and oversight questions
stat
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