The 13th European Conference on Rare Diseases and Orphan Products, held in Prague, Czech Republic, from June 3-4, 2026, served…
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How CTF is trying to rescue pharma’s shelved rare disease drugs
More than 5,000 potentially life-changing treatments for rare diseases are currently languishing on pharmaceutical company shelves, representing a vast reservoir…
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Unraveling the Mysteries of the Bundibugyo Virus: A Race Against a Rare and Deadly Outbreak in the Democratic Republic of Congo
As an Ebola outbreak grows in the Democratic Republic of the Congo, researchers are scrambling to learn more about the…
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Breakthrough Research Unveils How Gene Fusion Condensates Drive Rare Kidney Cancer Progression and Offers New Therapeutic Pathways
In a significant advancement for the field of oncology, a collaborative study led by investigators at the Johns Hopkins Kimmel…
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Rethinking evidence generation for ultra-rare diseases
Developing treatments for ultra-rare diseases presents fundamental challenges for traditional evidence generation, necessitating a paradigm shift in how scientific rigor…
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AbbVie Secures FDA Approval for Ultra-Rare Blood Cancer Therapy, While AstraZeneca Faces Delay for Breast Cancer Drug
The U.S. Food and Drug Administration (FDA) has recently issued a significant approval for AbbVie’s antibody-drug conjugate (ADC) targeting blastic…
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Johns Hopkins Researchers Uncover Liquid Condensate Mechanism Driving Rare Translocation Kidney Cancer and Propose New Therapeutic Avenues
In a significant advancement for the field of molecular oncology, a multidisciplinary team of investigators at the Johns Hopkins Kimmel…
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Rethinking evidence generation for ultra-rare diseases
Developing treatments for the approximately 7,000 known rare diseases, particularly the subset classified as ultra-rare, presents profound and often insurmountable…
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Breakthrough Research Unveils How Gene Fusion Condensates Drive Rare Kidney Cancer Progression and Potential Treatment Pathways
Investigators at the Johns Hopkins Kimmel Cancer Center and the Johns Hopkins Bloomberg School of Public Health have identified a…
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Rethinking Evidence Generation for Ultra-Rare Diseases
The landscape of drug development for ultra-rare diseases is undergoing a significant transformation, driven by the U.S. Food and Drug…
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