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Rare Disease · news · pharmadog
← back to Rare Disease
News · Rare Disease
840
stories
5d
STAT+: AstraZeneca invests $2 billion in Summit Therapeutics
stat
5d
STAT+: UniQure’s gene therapy continues to slow Huntington’s progression after four years
stat
5d
Opinion: Massive SNAP cuts could mean more children are separated from their families
stat
5d
Opinion: Child health is the national security crisis we keep ignoring
stat
5d
Why MAHA wants to make health data much more accessible
stat
5d
3 steps for taking on Novartis in radiopharma: Curium up first
biospace
6d
Egetis Therapeutics' Emcitate cleared by FDA for rare X-linked genetic disease
endpoints
6d
Recon: FDA approves Egetis’ Emcitate for rare genetic disease; Novo buys rights to Chinese weight-loss pill in $2.6B deal
raps
6d
FDA Approves First Treatment for MCT8 Deficiency
fda press
6d
Trump administration asks Supreme Court to allow transgender medical care restrictions in prison
stat
6d
An ‘off-the-shelf’ CAR-T therapy from Adicet shows promise against lupus
biopharma dive
6d
Nonprofits' gene therapy for rare immune disease will cost $3.25M
endpoints
6d
FDA nod for Mirum’s Atebrioz makes 3 options for ultra-rare bone disease
biospace
6d
Trump uses ‘contested’ power to cut more funding to HHS
stat
6d
Roche drops muscle-sparing obesity antibody after flunked interim analysis
fiercebiotech
6d
Opinion: AI is eroding the barriers that kept biological weapons rare
stat
7d
Milestone Sanfilippo nod helps Ultragenyx regroup after tough Angelman defeat
biospace
7d
Recon: FDA approves drugs for Parkinson’s, bile duct cancer, rare soft tissue disease; Merck, Daiichi scrap application for ADC ...
raps
9d
Trump administration uses rare authority to claw back nearly $1B in spending approved by Congress
stat
9d
FDA approves Mirum’s drug for ultra-rare soft tissue disease
endpoints
9d
To get drugmakers into ultra-rare disease, the answer isn’t rare disease at all
endpoints
9d
Adicet Bio CAR-T sparks lupus remissions in early trial as ‘new era’ of autoimmune treatment approaches
fiercebiotech
9d
Novo pursues long-acting obesity drugs; Acadia slumps on Alzheimer’s data
biopharma dive
9d
Federal judge rolls back landmark disability protections
stat
9d
Travere CEO Eric Dube on deciding to leave; Roche tees up a board seat for ex-Takeda chief
endpoints
9d
Opinion: RFK Jr.’s use of the National Center for Health Statistics will add confusion to measles deaths count
stat
10d
ADARx scores upsized $446M IPO, while fellow RNA biotech City maps out Nasdaq plans
fiercebiotech
10d
RNAi drug specialist ADARx raises $446M IPO
biopharma dive
10d
US senators grill FDA nominee Overton on ability to ‘stand up’ to Trump
biospace
10d
Elevar wins first FDA approval with second-line drug for bile duct cancer
endpoints
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