Scientists at the Johns Hopkins Kimmel Cancer Center and the Johns Hopkins Bloomberg School of Public Health have unlocked a…
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The FDA plans its third rare disease policy change in just over a year
On October 14, 2026, the U.S. Food and Drug Administration (FDA) formally unveiled the "Rare Disease Framework Initiative," a comprehensive…
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FDA Unveils Rare Disease Framework Initiative to Accelerate Novel Therapies and Streamline Drug Development
The United States Food and Drug Administration (FDA) has officially rolled out the Rare Disease Framework Initiative, a sweeping regulatory…
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Johns Hopkins Researchers Discover How Fusion Genes Drive Rare Kidney Cancer Through Cellular Droplets
Scientists at the Johns Hopkins Kimmel Cancer Center and the Johns Hopkins Bloomberg School of Public Health have unlocked a…
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FDA Approves Zanvastro as the First Disease-Modifying Treatment for Rare Alexander Disease
The U.S. Food and Drug Administration (FDA) has officially granted approval to Zanvastro, marking a watershed moment in medical history…
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Kilauea Volcano enters a rare era of frequent lava fountains as researchers uncover the mechanics of episodic eruptions
The iconic Kilauea volcano, long celebrated as one of the most active geological sites on the planet, has entered a…
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Shionogi Expands Rare Disease Portfolio With $2 Billion Acquisition of IntraBio and Flagship Drug Aqneursa
Japanese pharmaceutical major Shionogi has announced a definitive agreement to acquire IntraBio in a multibillion-dollar transaction valued at $2 billion.…
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Johns Hopkins Researchers Discover How Fusion Genes Drive Rare Kidney Cancer Through Liquid Droplet Formation
Scientists at the Johns Hopkins Kimmel Cancer Center and the Johns Hopkins Bloomberg School of Public Health have unlocked a…
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Atossa Therapeutics Unveils Shareholder Cash Reward Plan Tied to Rare Disease Priority Review Vouchers
Atossa Therapeutics has officially established a groundbreaking financial framework designed to directly reward its shareholders with a substantial cash payout…
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Johns Hopkins Researchers Uncover How Genetic Fusions Drive Rare Kidney Cancer Through Cellular Droplet Formation
Researchers at the Johns Hopkins Kimmel Cancer Center and the Johns Hopkins Bloomberg School of Public Health have published a…
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