Scientists have successfully restored hearing in animal models of ENPP1 deficiency, a genetic disease in which individuals ...

A study evaluating a pioneering lentivirus (LV)-mediated gene therapy trial for classical Fabry disease showed promising ...

What would you do to save your child? This mom is fighting to fund a cure for her daughter's terminal illness.

Sangamo Therapeutics Inc. (NASDAQ:SGMO) is one of the best penny stocks under $1 to buy now. On June 24, Sangamo Therapeutics ...

A researcher says an experimental gene therapy for a rare inherited disorder is saving almost as much money for the treatment of five patients as the study itself costs.

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A little 4 year old girl named Skylar is being treated right now at Primary Children’s Hospital for a rare, progressive and ...

The FDA has accepted for Priority Review the BLA for tividenofusp alfa for the treatment of Hunter syndrome, also known as MPS II.

The specialist said that conventional enzyme-replacement therapy has to occur every two weeks, requiring approximately two hours for each treatment.

The global Enzyme Replacement Therapy (ERT) market is poised for significant expansion, projected to reach USD 21.62 billion ...