Posts

Garijo’s Plan to Revamp Sanofi: R&D Cleanup, More M&A, and a China Push Explained

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RA Capital Launches Rare Disease Company Oak Hill Bio onto Nasdaq via SPAC

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AZ stays on track for $80B revenue goal as cancer meds, rare disease sales help offset Ultomiris miss

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Sarepta Taps Former AbbVie and Tessera CEO Michael Severino as New CEO to Lead Duchenne Drugmaker's Turnaround

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Broad Institute launches Center for Therapeutic Genetics for rare diseases

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Recent News: ARPA-H Awards Up to $160M for Custom Genetic Medicines

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Vertex Inks $10B Crinetics Buy, Entering White Space Blockbuster Opportunity

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Through Flux and Fluidity, FDA Keeps Coming Back to Rare Disease: 2026 Policy Shifts and Approvals in Rare Disease Space

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FDA Approvals Outrun Staff Cuts: 23 Novel Drugs Mark Best First Half in Three Years

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Ipsen signals more deals to continue buying spree

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Insilico lands heavily backloaded $2.5B AI drug discovery deal with SK Biopharm

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Busy FDA gives rare disease sector complementary pathways, unanswered questions – 2026 snapshot

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Travere pays Everest $112.5M upfront to grow rare kidney disease pipeline

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BioMarin Suffers Another Blow to Rare Disease Portfolio in Phase 3 Flop

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Zydus Lifesciences to Pursue Acquisitions in Specialty and Rare Diseases

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BioMarin's rare disease therapy shows no clinical benefit in Phase 3 test

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Angelini Fortifies Neurology Portfolio with $4.1B Buyout of Catalyst

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Consequences of Alternative Funding Programs for Specialty Drugs: Patient Risks and Industry Concerns

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Small Pools, Big Ideas: How the Rare Disease Community is Reimagining Clinical Trials

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Travere Therapeutics Receives FDA Approval for Filspari as First FSGS Treatment

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