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Rare Disease · news · pharmadog
← back to Rare Disease
News mentioning Rare Disease
220
stories
7d
PTC takes a chance on Sangamo’s Fabry disease gene therapy
biopharma dive
7d
STAT+: Eli Lilly targets booming black market for retatrutide
stat
7d
Bankrupt Sangamo offloads assets to Lilly, PTC in $161M sale
biospace
7d
STAT+: Neurocrine Biosciences and the delicate balance of benefit and risk
stat
8d
Deaths reported after treatment with Neurocrine’s approved Prader-Willi drug
biospace
8d
STAT+: Pharmalittle: We’re reading about Lilly ‘black market’ lawsuits, pushback over Trump vaccine plan, and more
stat
8d
STAT+: Definium Therapeutics says its LSD therapy eased anxiety in late-stage trial
stat
8d
STAT+: Biotech company co-founded by top autoimmune researcher lays out plans with antibody therapies
stat
9d
STAT+: Inside a $7 billion Silicon Valley startup’s mad dash to automate the business of health care
stat
9d
STAT+: Neurocrine Biosciences rare disease drug possibly tied to safety issues, experts say
stat
10d
Jazz commits up to $1.3B to buy Actio, bolster epilepsy pipeline
biospace
10d
STAT+: Sionna’s experimental cystic fibrosis pill falls flat in mid-stage trial
stat
10d
STAT+: Silence Therapeutics aims at Takeda with new data in rare blood cancer
stat
10d
BioMarin axes asset from $270M Inozyme takeover, ending run in rare metabolic indication
biospace
13d
BioMarin discontinues rare disease drug; CSL and Arcturus split up
endpoints
13d
Ensoma sheds more staff, pauses preclinical programs to home in on lead gene therapy
fiercebiotech
14d
BioMarin axes $270M rare disease asset after missing phase 3 endpoint
fiercebiotech
14d
Third time’s the charm for Replimune as melanoma drug earns FDA greenlight
biospace
14d
Opinion: One year since Kennedy canceled mRNA vaccine projects, science shows how wrong he was
stat
15d
CROs vs. biotechs: Finding the right fit
biospace
15d
FDA approves Moderna’s mRNA flu vaccine, the first to use the technology
stat
15d
STAT+: Takeda’s narcolepsy drug approved by the FDA, seen as a boon for new class of treatments
stat
15d
STAT+: Federal regulators invite industry, researchers, and lobbyists to closed-door meetings on clinical AI
stat
15d
Erica Schwartz confirmed as CDC director, filling nearly yearlong vacancy
stat
15d
STAT+: Could poop pills be the next treatment for food allergies?
stat
15d
STAT+: Once again, child dies in gene-editing trial in China, rekindling debate on transparency and safety
stat
15d
Bespoke gene-editing outfit abandons lead program, cuts ‘several’ employees
biospace
15d
Chaotic adcomms threaten to derail FDA’s bid to renew trust after Makary, Prasad
biospace
15d
STAT+: Moderna launches early trial of Ebola mRNA vaccine
stat
15d
STAT+: Pharmalittle: We’re reading about a Medicare pilot and orphan drugs, Pfizer cutbacks, and more
stat
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