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Rare Disease · news · pharmadog
← back to Rare Disease
News · Rare Disease
856
stories
May 30, 26
STAT+: China competition, ‘destruction’ at FDA give agency chance to restructure, Rick Pazdur says
stat
May 29, 26
Kenya court suspends U.S. plan for Ebola quarantine facility for Americans
stat
May 29, 26
Agios ends development of tebapivat in low-risk MDS following subpar results
endpoints
May 29, 26
Allogene CEO to step down; Agios drug fails key test
biopharma dive
May 28, 26
AbbVie clinches FDA approval for ultra-rare blood cancer ADC from ImmunoGen purchase
endpoints
May 22, 26
FDA Approves First Treatment for Chronic Hepatitis Delta Virus (HDV) Infection
fda press
May 19, 26
How AI helped treat a newborn’s ultra rare disease. ‘It was almost like a light switch.’
stat
May 19, 26
BioMarin drug acquired in buyout misses goal in rare disease study
biopharma dive
May 19, 26
STAT+: A new ranking of pharma companies by R&D performance
stat
May 19, 26
Relay drug shows early promise against rare blood vessel diseases
biopharma dive
May 19, 26
Relay's PI3Kα inhibitor clears efficacy bar in Phase 2 vascular anomalies study
endpoints
May 19, 26
BioMarin's rare disease therapy shows no clinical benefit in Phase 3 test
endpoints
May 18, 26
Amgen stands by rare disease drug Tavneos amid Japan liver toxicity report
endpoints
May 18, 26
Høeg fired in latest FDA shakeup; 20 people die after taking Amgen drug
biopharma dive
May 18, 26
Texas is opening up a detransition clinic
stat
May 18, 26
Opinion: ‘Patient autonomy’ has nothing to do with childhood vaccine policies
stat
May 18, 26
Opinion: How to restore credibility to the CDC’s vaccine advisory committee
stat
May 17, 26
In Ebola outbreak, a number of Americans in the Congo believed to have had exposure to suspected cases
stat
May 17, 26
STAT+: The hantavirus outbreak is prompting Covid flashbacks — including the conspiracies
stat
May 16, 26
Opinion: Marty Makary misunderstood something fundamental about the FDA
stat
May 15, 26
STAT+: FDA leadership shakeup widens following Makary’s departure
stat
May 15, 26
Biotech leaders campaign for Pazdur; Takeda to lay off 4,500 workers
biopharma dive
May 15, 26
Opinion: First hantavirus, now Ebola: What two outbreaks reveal about global preparedness
stat
May 15, 26
Aardvark's next move after clinical hold; Alumis' verdict on Acelyrin asset
endpoints
May 15, 26
A new Ebola outbreak is confirmed in a remote Congo province, with 65 deaths recorded
stat
May 15, 26
#ASGCT26: A Zillow-like marketplace for abandoned gene therapies goes live
endpoints
May 15, 26
STAT+: Top U.S. officials pressured Germany to pay more for prescription drugs
stat
May 14, 26
Makary’s departure and Cassidy’s tenuous Senate seat
stat
May 14, 26
Imaging Clues That Raise Suspicion for Diffuse Midline Glioma
onclive
May 14, 26
With new data, Regenxbio to seek FDA approval of Duchenne gene therapy
biopharma dive
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