UK health system to offer cutting-edge gene therapy for sickle cell disease
Britain's National Health Service (NHS) will provide a cutting-edge gene therapy that aims to cure sickle cell disease, the ...
Gene therapy may be 'one-shot stop' for rare bone disease
For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...
Gene therapy and bone marrow transplant alleviate ultra-rare genetic disease symptoms
In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and ...
Noida’s child PGI detects rare gene defect in a baby, docs say India’s 2nd case
Noida: Doctors at Noida's Post Graduate Institute of Child Health have detected a rare Coenzyme Q10 deficiency in a ...
Turning Tragedy into Action: A Family's Fight for Genetic Awareness
When we can play any part in supporting other families, it’s a tragic gift that allows us to continue to parent Havi.” For ...
openaccessgovernment1d
Gene therapy offers new hope for sickle cell disease patients
A new gene editing treatment has been approved for use in the NHS, which will help treat sickle cell disease patients ...
‘Ground-breaking’ gene treatment for sickle cell disease to be rolled out on NHS
A GROUNDBREAKING genetic treatment with the potential to cure sickle cell disease will be rolled out on the NHS. The one-off ...
The Columbian46m
Study: Genetic diversity falling
Two-thirds of animal and plant populations are declining in genetic diversity, which makes it harder to adapt to environmental changes, according to research published Wednesday.
Spur Therapeutics Announces Successful End-of-Phase 2 Meeting with FDA for FLT201, Its Gene Therapy Candidate for Gaucher Disease
FDA aligned on single-arm Phase 3 study to support potential accelerated and full approval On track to dose first patient in Phase 3 trial in second half of 2025 LONDON, Feb. 03, 2025 (GLOBE NEWSWIRE) ...
NHS to offer 'groundbreaking' sickle cell gene therapy
The gene therapy Casgevy, which will now be offered to some patients in England, could help people live disease-free, experts ...
National Institute for Health and Care Excellence4d
Groundbreaking one-off gene therapy approved for severe sickle cell disease
People in England to benefit from groundbreaking one-off gene therapy for severe sickle cell disease approved by NICE ...