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Business
FDA offers new details on quicker approval pathway for personalized gene therapies
12 news sources are covering this Business story right now — PULSE is tracking how fast it spreads.
Rare Diseases
8 trends tracked about this subject — the full history, oldest to newest below.
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Health
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World-first surgery for baby's rare skull condition
A one-year-old baby has undergone a world-first surgery utilizing super-elastic springs to treat a rare condition involving a fused skull.
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Business
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RK Mellon Foundation pledges $25M to launch rare disease therapy hub in Pittsburgh - Pittsburgh Post-Gazette
An accelerator backed by the RK Mellon Foundation aims to tackle rare diseases lacking approved drugs.
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Health
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America Getting Lesson in Consequences of Trump’s War on Public Health as Rare Disease Outbreaks Ravage the Country
U.S. public health faces severe tests from rare disease outbreaks amid funding cuts and vaccine resistance.
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Health
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Targeting Allele-Specific Faulty MRNA In SCNA2 Mutation Patients
Personalized gene therapy using bespoke ASOs has enabled a teen with SCN2A-related epileptic encephalopathy to walk independently for the first time.
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Business
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Unpacking Anthropic's 100-day sprint into biopharma: Nobel hires, M&A and major ambition
Anthropic is aggressively entering the biopharma sector through a 100-day sprint involving Nobel laureates, M&A activity, and new scientific AI tools.
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Health
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$160 million ‘moonshot’ backs Boston effort to revolutionize rare disease treatment
A $160 million non-profit initiative in Boston aims to revolutionize the development of genetic medicines for rare and ultra-rare diseases.
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Entertainment
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Former wrestler Tyler Mane says he was diagnosed with breast cancer, urges men to get checked
Hollywood icon Tyler Mane’s breast cancer diagnosis sparks rare male health awareness