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Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in ...

Announcing a new article publication for BIO Integration journal. Neurologic disorders currently affect approximately 100 ...

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

Australia 2 Gene Therapy Research Unit, The Children’s Hospital, Westmead and Children’s Medical Research Institute, Sydney, NSW, Australia Correspondence to: Professor Trent Department of Molecular ...

The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

Researchers have identified a method to enhance the effectiveness of a promising cancer treatment. They found that modifying a specific gene improves the ability of immune cells to combat cancer for ...

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.

The National Institute for Health and Care Excellence (NICE) has approved a one-off gene editing therapy to treat severe sickle cell disease (SCD) in people 12 years and over. In final draft guidance1 ...

Exagamglogene autotemcel (exa-cel) gene therapy offers a potential cure for severe sickle cell disease, now available on NHS.

The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years ...