Farrell High School students are raising awareness for an important cause--donating blood for sickle cell anemia.
Actor turned rare disease advocate Luke Rosen tells how his daughter, Susannah, lives with a KIF1A-associated neurological ...
Patients with CaV2.1 channelopathies face severe and often debilitating symptoms, such as seizures, migraines, tremors, and ...
Mayo Clinic researchers have developed a promising gene-editing therapy that directly corrects a genetic mutation responsible for autosomal dominant polycystic kidney disease (ADPKD), the most common ...
In November 2023, the UK became the first country in the world to authorise a Crispr gene-editing therapy.
Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...
A developmental screening tool may identify young children with SCD who are at risk for academic difficulties, a study found.
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How sickle cell changed human evolution

Sickle cell disease is caused by a single genetic mutation that alters the shape of red blood cells. While the condition ...
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