rare diseases
4 stories
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healthFDA Draft Guidance Aims to Speed Up Genome Editing Therapies
The FDA proposes letting companies use existing data for genome therapies, aiming to speed development for rare diseases.
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healthMarathoners Race for Rare Disease Awareness
Athletes leverage marathon training and race participation to shed light on underrepresented rare diseases, driving fundraising and support for affected communities.
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healthTakeda Restructures to Accelerate Late-Stage Drug Pipeline
Takeda launches a major restructuring to cut $1.25B in costs by 2028 and redirect savings into R&D for oncology, neuroscience, and rare diseases.
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healthRare Childhood Lung Disease Severely Impacts Quality of Life
New research reveals hospitalizations and growth impairment significantly reduce quality of life for children with rare interstitial lung diseases.