New research found that gene therapy for children with a form of severe combined immunodeficiency was successful in 95% of ...

Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the ...

An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has ...

A significant advance in treatment of patients with immunity to AAVs previously ineligible for clinical trials and existing ...

The mucopolysaccharidosis (MPS) market is rapidly evolving due to rising demand for effective therapies to manage glycosaminoglycan buildup and improve patient quality of life. Enzyme Replacement ...

Preclinical study showed PDIA1 and PDIA5 protect cells from oxidative stress, maintain mitochondrial function, and are involved in androgen receptor regulation.

Xanthine oxidase inhibitors, primarily allopurinol and febuxostat, are the mainstay of treatment. The goal of chronic gout management includes lowering serum urate levels to below 6 mg/dL. 5 Overall, ...

Please provide your email address to receive an email when new articles are posted on . Researchers analyzed 19 children with Pompe disease seen at a Texas-based children’s hospital.

It’s a big day for Sanofi as the pharmaceutical giant welcomes two European drug approvals, both for rare enzyme deficiencies. One regulatory nod ushers in the first treatment in Europe for a ...

Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window The first test ...