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Rare Disease · news · pharmadog
← back to Rare Disease
News mentioning Rare Disease
225
stories
Jun 1, 26
STAT+: Servier to buy Edgewise Therapeutics’ muscular dystrophy drug
stat
Jun 1, 26
2026 Orphan Drugs Report: A safe(r) passage amid stormy waters?
biopharma dive
May 29, 26
Allogene CEO to step down; Agios drug fails key test
biopharma dive
May 19, 26
How AI helped treat a newborn’s ultra rare disease. ‘It was almost like a light switch.’
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May 19, 26
BioMarin drug acquired in buyout misses goal in rare disease study
biopharma dive
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 15, 26
Aardvark's next move after clinical hold; Alumis' verdict on Acelyrin asset
endpoints
May 15, 26
STAT+: Top U.S. officials pressured Germany to pay more for prescription drugs
stat
May 14, 26
With new data, Regenxbio to seek FDA approval of Duchenne gene therapy
biopharma dive
May 14, 26
STAT+: Regenxbio says Duchenne gene therapy succeeded in clinical trial, paving way for FDA submission
stat
May 13, 26
FDA’s leadership void leaves biotech with renewed ‘uncertainty’
biopharma dive
May 13, 26
Abeona pivots to T cells; AstraZeneca's rare disease drug passes Phase 3
endpoints
May 13, 26
STAT+: Drug meant to make gene therapy safer may also make it less effective
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May 13, 26
STAT+: Scientists link boy’s tumor to gene therapy viruses, in rare finding
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May 12, 26
STAT+: Why Marty Makary was the worst FDA commissioner in 25 years
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May 12, 26
STAT+: Makary departs FDA amid turmoil as Diamantas, agency’s top food official, steps in
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May 12, 26
STAT+: Hims’ sales miss as telehealth competition grows
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May 12, 26
STAT+: Capsida says it still doesn’t know what caused gene therapy death
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May 12, 26
A year after Vertex’s big launch, pain drug research faces a pivotal moment
biopharma dive
May 11, 26
No FDA permission, no problem: New flavored vape policy worries experts
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May 11, 26
STAT+: Astellas treatment offers new hope to a devastated rare disease community
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May 11, 26
STAT+: Five years after disaster, a rare disease community gets new chance at treatment
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May 11, 26
STAT+: Medicare is spending far less than expected on new Alzheimer’s drugs
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May 11, 26
Animal skin disease confirmed in clusters of European men who have sex with men
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May 8, 26
STAT+: The biotech news you need to read today
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May 7, 26
STAT+: FDA revisits a rare cancer treatment it rejected a few months ago
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May 7, 26
STAT+: FDA to reconsider treatment for rare cancer after its surprise rejection
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May 7, 26
STAT+: Next-gen Duchenne drug from Entrada disappoints
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