Explore News
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Japan’s Takeda Warns Against Rare Disease Drug After 20 Deaths
Takeda Pharmaceutical halts new prescriptions of a drug for rare autoimmune diseases after 20 patient…
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New Gene Therapy Enables Children With a Rare Form of Deafness to Hear
A groundbreaking gene therapy brings hearing to children with a rare form of deafness
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AI Adoption Surges in Rare Disease Management
Elevating patient voices in AI-driven rare disease management
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1 in 2,500 Children Affected by Rare Disease
Sophia’s heart-wrenching story sheds light on this rare condition




