With a shared purpose, we can realize the true promise of CRISPR and improve healthcare, providing hope to more patients with ...
Researchers led by the University of Vienna and Liège University Hospital Center have identified genetic variants associated ...
Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunru crafted a bespoke treatment that has successfully corrected the genetic ...
Alström syndrome affects roughly one in 500,000 people. In May, Fort Worth will host a symposium of researchers looking into treatments.
Over the next two years, the planned funding will be allocated to projects that have received some FDA feedback as well as emerging efforts.
A "gene silencer" (technically known as small interfering RNA, or siRNA), locally delivered by nanoparticles embedded in an ...
AMD is one of the rarest documented genetic disorders. Only around 10 million people in the world carry the altered NPR2 gene, and of those, just 3,500 are estimated to display the physiological ...
When two defective gene variants combine, normal protein function can sometimes be restored.
The crisis facing rare disease and disability families in Connecticut is caused by systemic underinvestment, administrative barriers, and policy choices.
A single, untargeted proteomics test for rare genetic diseases has been developed. A research team from the University of Melbourne (Australia) and Murdoch Children’s Research Institute (Victoria, ...
Sinar Daily on MSN
Roberts syndrome: A rare genetic disorder
The number of cases in Malaysia is very small. However, the risk still exists, particularly for parents with a family history ...
When you have a rare disease, managing doctor's appointments, prescriptions and care plans is difficult enough — figuring out what Medicare will (and won't) cover often adds another layer of stress.
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