In a landmark study published in the journal Cell Reports on April 22, investigators at the Johns Hopkins Kimmel Cancer…
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FDA Proposes Permanent Authorization of Rare Pediatric Disease Priority Review Voucher Program to Foster Regulatory Stability
The United States Food and Drug Administration (FDA) is making a significant move to permanently authorize the rare pediatric disease…
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Johns Hopkins Researchers Uncover Liquid Condensate Mechanism Driving Rare Translocation Renal Cell Carcinoma and Potential Therapeutic Pathways
In a significant advancement for the field of molecular oncology, a multidisciplinary team of investigators at the Johns Hopkins Kimmel…
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Biogen Acquires Apellis Pharmaceuticals for $5.6 Billion to Bolster Immunology and Rare Disease Portfolio
Biogen, a global biotechnology company renowned for its pioneering work in neuroscience, has announced a significant strategic acquisition of Apellis…
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Rocket Pharmaceuticals Secures Landmark FDA Approval for Kresladi, Ushering in a New Era for Ultra-Rare Immune Disease Treatment
The U.S. Food and Drug Administration (FDA) has granted accelerated approval to Rocket Pharmaceuticals’ Kresladi (marnetegragene autotemcel), a groundbreaking gene…
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Johns Hopkins Researchers Uncover Molecular Mechanism Behind Rare Kidney Cancer Driven by Fusion Gene Liquid Droplets
In a significant advancement for the field of molecular oncology, a multidisciplinary team of investigators at the Johns Hopkins Kimmel…
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A Rare Lunar Impact Creates a Crater the Size of Two Football Fields
The moon, a silent sentinel in our night sky, has revealed a dramatic new feature: a fresh impact crater, estimated…
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New drug cuts seizures by up to 91% in children with rare epilepsy
In a landmark development for pediatric neurology, an international clinical trial has demonstrated that an experimental genetic therapy, zorevunersen, can…
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Johns Hopkins Researchers Uncover Molecular Mechanism Behind Rare Kidney Cancer Progression via Liquid Condensates
In a significant advancement for the field of molecular oncology, a collaborative study led by investigators at the Johns Hopkins…
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