UMD researchers have discovered key mechanisms in gene regulation that could improve the design of RNA-based medicines.

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three-to-six times each week.

A GROUNDBREAKING genetic treatment with the potential to cure sickle cell disease will be rolled out on the NHS. The one-off treatment costs more than £1million per patient but may essentially ...

Our independent appraisal committee has approved the use of gene editing therapy exagamglogene autotemcel (exa-cel) for use in the NHS in England, providing a potential cure for some people with ...

This article is part of a Health Affairs Forefront series on “The Future of Sickle Cell Disease Research ... innovative treatment options. Both bone marrow transplants and gene therapy require ...

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Would it be a cure for my sickle cell disease? It was a decision I could think about making because my sister was a genetic match and could donate her stem cells. Most with sickle cell don’t ...

A sickle cell disease “cure” is to be offered to NHS patients for the first time. The £1.65 million gene therapy called exa-cel, also known under the brand name Casgevy, uses a Nobel Prize ...

There's new hope for couples who have discovered a genetic disease that may affect their future children. Doctors in the UAE are pioneering new methods that will allow them to treat a genetic ...