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 ...
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 ...
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 ...
EurekAlert!7d
JAX researchers alleviate symptoms of ultra-rare genetic disease
In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches -- gene therapy ...
uniQure advances gene therapy trial for Fabry disease
LEXINGTON, Mass. - uniQure N.V. (NASDAQ: NASDAQ:QURE), a gene therapy company with a market capitalization of $767 million, ...
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 ...
‘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 ...