More than 5,000 potentially life-changing treatments for rare diseases are currently languishing on the shelves of pharmaceutical companies, dormant and…
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Breakthrough in Rare Kidney Cancer Research Reveals How Gene Fusions Drive Tumor Progression via Liquid Condensates
In a significant advancement for the field of molecular oncology, a multi-disciplinary team of investigators from the Johns Hopkins Kimmel…
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Mapping the Foundations of Human Life First Blueprint of Skeletal Development Unveils Origins of Arthritis and Rare Bone Conditions
In a landmark achievement for regenerative medicine and developmental biology, an international consortium of researchers has published the first comprehensive…
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How CTF is trying to rescue pharma’s shelved rare disease drugs
Across the global pharmaceutical landscape, an estimated 5,000 potential treatments for rare diseases are currently languishing on corporate shelves, representing…
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Scientists Identify Liquid Condensate Mechanism Driving Rare Kidney Cancer and Potential New Treatment Pathways
A collaborative research effort led by scientists at the Johns Hopkins Kimmel Cancer Center and the Johns Hopkins Bloomberg School…
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Johns Hopkins Researchers Uncover Molecular Mechanism Driving Rare Translocation Kidney Cancer Through Liquid Condensate Formation
In a significant advancement for the field of oncology, investigators at the Johns Hopkins Kimmel Cancer Center and the Johns…
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How CTF is trying to rescue pharma’s shelved rare disease drugs
Across the global pharmaceutical landscape, a staggering number of potentially life-changing therapies for rare diseases lie dormant, gathering dust on…
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Navigating the Diagnostic Labyrinth: Fragmentation Hinders Rare Disease Progress Despite Scientific Leaps
The journey from the first symptom of a rare disease to a confirmed diagnosis, often referred to as the "diagnostic…
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Europe Must Rethink Funding and De-Risk Assets to Secure its Rare Disease Research Pipeline
The 13th European Conference on Rare Diseases and Orphan Products, held in Prague, Czech Republic, from June 3-4, 2026, served…
Read MoreHow 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…
Read More
