Texas Children’s has for the first time used a new enzyme replacement therapy called Kebilidi as a cure to AADC, a rare neurological disorder.

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.

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 ...

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

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, ...

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 ...

An international study has uncovered a new vulnerability in prostate cancer cells that could help improve treatment for one ...

Please provide your email address to receive an email when new articles are posted on . A phase 3 trial evaluating intrapleural enzyme therapy vs. video-assisted thoracoscopic surgery is feasible.

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 ...

Scientists at UC Irvine have found a way to potentially reverse age-related vision loss by targeting the ELOVL2 “aging gene” ...

Prostate cancer is the second most common cancer in men worldwide. The post Docs find new vulnerability that may mean better ...