Ongoing clinical research at UNC could lead to a first-of-its-kind enzyme replacement therapy for Hunter syndrome, an ...

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.

Please provide your email address to receive an email when new articles are posted on . Mean maximum ADAMTS13 activity after recombinant ADAMTS13 exceeded 100%. Markedly fewer patients receiving ...

When CEO Paul Hudson took over at Sanofi in 2019, he launched a “play to win” strategy centered on first-in-class and best-in-class drugs in therapeutic areas where the company was lacking. On Tuesday ...

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 ...

A study evaluating a pioneering lentivirus (LV)-mediated gene therapy trial for classical Fabry disease showed promising results over five years, indicating a potential breakthrough in treatment for ...

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 ...

Tividenofusp alfa treatment showed reduction and normalization in key disease biomarkers, stabilization or improvement in clinical endpoints ...

Breakthrough Therapy Designation Supported by Integrated Long-Term Clinical Data Demonstrating Normalization in Cerebral Spinal Fluid Heparan Sulfate Non-Reducing End (CSF HS-NRE) U.S. FDA Confirmed ...

Denali Therapeutics announced that results from its open-label Phase 1/2 trial of tividenofusp alfa for Hunter syndrome were published in the January 1, 2026 issue of The New England Journal of ...

AB-1009 is an investigational gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated. For more information, ...

In a recent study posted to the medRxiv* preprint server, researchers evaluated the impact of the coronavirus disease 2019 (COVID-19) pandemic on the rates of pancreatic enzyme replacement therapy ...