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The exceptionally rare disease that causes holes to form in your brain
Creutzfeldt-Jakob disease is an extremely rare and fatal brain-wasting disease that's like a human version of "mad cow." ...
She faced major hurdles in her rare disease fight. These barriers can cost lives, experts say
When Madi Vanstone was 11, she found a medication that would be life changing for her battle against cystic fibrosis, but it ...
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Portugal. The Man highlight rare disease month as bandmember’s daughter battles genetic disease
February is Rare Disease Month, an annual effort to highlight the challenges families can face while navigating rare ...
Activating complex regions of the genome to treat rare diseases: Researchers identify a a master epigenetic switch
Biomedical engineers at Duke University have demonstrated a promising new approach that could be used to treat a rare and ...
Acsh.org3d
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 ...
Why Is Rare Disease Focused Ascendis Pharma Stock Trading Higher On Thursday?
Ascendis Pharma beat Q4 earnings and revenue estimates, driven by a $100M Novo Nordisk deal and Yorvipath's EU launch.
Young girl with rare disease looking for first responder patches for her lengthy road to recovery
A young girl with a rare disease is preparing for a lengthy road to recovery. She’s hoping to decorate her hospital room with ...
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 ...
EurekAlert!5d
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 ...
‘Noah,’ a Documentary About a Woman With Seven Rare and Incurable Diseases, Acquired by Jambika Docs (EXCLUSIVE)
The film follows Noah, a 26-year-old activist, who has turned her battle against seven rare and incurable diseases into a ...
Science Daily1d
Gene variant could protect against ultra-rare seizure disorder, fruit fly research finds
A combination of human pedigree analysis and fruit fly genetics has identified a gene variant that appears to prevent PIGA-CDG, a rare but serious seizure disorder. The findings could ultimately lead ...