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Rare Disease · news · pharmadog
← back to Rare Disease
News mentioning Rare Disease
345
stories
11d
Chutes & Ladders—Travere chief makes way for former Amicus CEO
fiercebiotech
11d
Kyverna steps closer to FDA-first CAR T for an autoimmune condition with durability data
biospace
11d
Roche pens backloaded $2B deal with Atavistik to discover allosteric CVRM drugs
fiercebiotech
12d
Baby KJ's gene editing drug cost $1.65M to make, Fyodor Urnov says
endpoints
12d
Ionis-Otsuka offer 'groundbreaking' improvement in function and survival for genetic type of ALS
biospace
12d
Novo’s turnaround plan, weight loss wins, Ultragenyx’s Sanfilippo approval, IPO blitz
biospace
13d
Viking achieves 'maintenance trifecta' as GLP-1/GIP dual agonist exceeds expectations
biospace
13d
Lilly presents early data supporting GLP-1/GIP candidate's potential in depression, alcohol use disorder
biospace
13d
Otsuka heads to FDA as Ionis-partnered ALS drug hits primary phase 3 endpoint
fiercebiotech
14d
STAT+: What a vote on Grail’s cancer detection test could mean for the company, patients, and regulators
stat
14d
STAT+: Lilly’s breast cancer combo wins approval
stat
14d
STAT+: Alkermes’ orexin agonist shows potential to treat ADHD in early-stage trial
stat
14d
Mikhail, Davis on board with regulatory framework tailored to rare disease therapies
biospace
15d
STAT+: Radiopharma companies merge after one receives surprise FDA rejection
stat
17d
This week in charts: GSK’s China deals, the $300M IPO club and selling the news
biopharma dive
17d
STAT+: Roche expands its Boston footprint with new research center
stat
18d
Electra amasses $350M in biotech’s latest big IPO
biopharma dive
18d
STAT+: FDA approves a new gene therapy for Sanfilippo syndrome, an ultra-rare disease
stat
18d
FDA approves Ultragenyx's gene therapy for Sanfilippo syndrome type A
endpoints
18d
Chutes & Ladders—Legend poaches Novartis vet to boost commercial potential
fiercebiotech
18d
After Novartis’ devastating M&A losses, long live the executive willing to try
biospace
19d
Deep dive: Psychedelics back on FDA’s doorstep
biospace
19d
Novartis halts TREM2 targeting ALS program in 4th trial stumble in a month
biospace
19d
FDA hears input on psychedelics, PDUFA, lung cancer’s next-gen therapies boost survival
biospace
19d
AI is changing the talent biotech companies compete to hire
biospace
19d
5 biotechs braving the commercial waters with product launches
biospace
20d
FDA opens Operation TrialBlazer for applications, hoping to reshore early trials
biospace
20d
Rare disease reform, CMC risk, reshoring US trials top of mind ahead of PDUFA VIII meeting
biospace
21d
FDA looks into reassessing criteria around rare disease trials, endpoint selection
endpoints
21d
Definium moves toward FDA submission for LSD drug in anxiety after ‘another clear win’
biospace
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