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Rare Disease · news · pharmadog

← back to Rare Disease

News · Rare Disease

856 stories

Apr 27, 26Q&A: Intellia CEO talks first Phase 3 CRISPR readout and what’s nextendpoints

Apr 27, 26STAT+: Astellas retries XLMTM gene therapy after deathsstat

Apr 27, 26STAT+: Pharmalittle: We’re reading about a Lilly deal, an Intellia CRISPR rare-disease treatment, and morestat

Apr 27, 26Intellia CRISPR drug succeeds in late-stage study against rare swelling disorderbiopharma dive

Apr 27, 26STAT+: Intellia says CRISPR-based treatment for rare disease reduced swelling attacks in pivotal trialstat

Apr 27, 26STAT+: Zap in a cap: How one neurotech startup is using a hat to treat depressionstat

Apr 24, 26International childhood vaccine campaign growsstat

Apr 24, 26Opinion: I started medical school at 69 and will begin residency at 72. Here’s what I learnedstat

Apr 23, 26STAT+: Trump celebrates closing first round of drug pricing deals, promises more aheadstat

Apr 23, 26FDA approves Regeneron’s hearing loss gene therapybiopharma dive

Apr 23, 26FDA approves Regeneron's hearing loss gene therapy Otarmeniendpoints

Apr 23, 26Heart’s beat may help it beat cancer, mouse research suggestsstat

Apr 23, 26STAT+: Legislatures in Colorado and Virginia resist moves to constrain drug affordability boardsstat

Apr 23, 26Sanofi posts upbeat sales as R&D pressure buildsbiopharma dive

Dec 23, 25Fierce Biotech Layoff Tracker 2026: Foghorn cuts workforce by 40%; BioMarin drops 117 rolesfiercebiotech

Dec 22, 25Fierce Biotech Fundraising Tracker '26: TriGemX tacks on $94M; AnaCardio adds $71Mfiercebiotech

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