An investigational gene therapy showed sustained clinical efficacy for children born with ADA-SCID. In a study of 62 children, overall survival after treatment was 100% and event-free survival was 95% ...

An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has restored and maintained immune system function in 59 of 62 children born ...

Adenosine deaminase (ADA) deficiency is a rare autosomal recessive disorder that underpins a severe form of combined immunodeficiency (SCID), resulting in the accumulation of toxic metabolites that ...

Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the long-term safety and efficacy of the curative treatment, in a study led by ...

Takeaway Gene corrected stem cell therapy represents a major scientific breakthrough for ADA SCID. When children are diagnosed early and treated quickly they can develop their own strong immunity and ...

DUBLIN – Bidding to become the second European firm to win a gene therapy approval, Glaxosmithkline plc filed a marketing authorization application (MAA) with the EMA for GSK2696273, a treatment for ...