Gene variant could protect against ultra-rare seizure disorder, fruit fly research finds
One in 10 Americans live with a rare disease, which often lacks effective treatments. Such is the case for PIGA-CDG, an ultra ...
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8-year-old in Stokes County fights rare brain disease
We designed our dream home in Mexico for $270,000. It was cheaper than buying in the US but came with extra costs we didn't ...
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Rare Diseases Are Neglected. The FDA Needs A Dedicated 'Center of Excellence for Rare Diseases'.
The FDA has made some modest efforts, but it needs an internal organization that understands and would be an aggressive ...
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Texarkana boy battling rare disease prepares for life-changing transplant
TEXARKANA, Texas - A Texarkana, Texas boy battling a rare genetic disease is preparing to head to Arkansas Children's ...
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Amid takeover talk, SpringWorks scores FDA nod for rare genetic disease treatment Gomekli
Tuesday, the agency signed off on SpringWorks’ Gomekli (mirdametinib) as a treatment for the rare genetic disorder ...
AI tool helps find life-saving medicine for rare disease
After combing through 4,000 existing medications, an artificial intelligence tool helped uncover one that saved the life of a ...
‘Noah,’ a Documentary About a Woman With Seven Rare and Incurable Diseases, Acquired by Jambika Docs (EXCLUSIVE)
Barcelona-based sales agency Jambika Docs has acquired the documentary feature “Noah” ahead of the European Film Market in ...
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Uncovering two rare infectious diseases which might be responsible for COVID-19
In a landmark discovery, researchers applied a novel AI model called the max-logistic intelligence classifier to analyze ...
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Public health strategies skip patients with rare diseases, says UK study
A study led by UCL found that individuals with rare diseases were five times more likely to die from Covid-19 than the ...
Delay in roll out of ₹974 crore under rare diseases policy hits patients
Advocacy groups urge Health Ministry to intervene urgently in delayed funding for rare disease treatments, impacting patients ...
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AI approach reveals possible fusion of rare diseases in COVID-19 origins In A Landmark Dis...
Despite extensive research, the origins of COVID-19 remain elusive. In a new study published in the KeAi journal Advances in Biomarker Sciences and Technology (ABST), an AI-driven approach was adopted ...
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What happens when a rare disease drug seems to work, but the health care system doesn’t?
Cristina Brennan can hear the clock ticking. At the end of the year, her 12-year-old son Tristan will no longer have free ...