The therapy shows efficacy in improving diarrhea, abdominal pain in GLP-1, GLP-1/GIP-treated patients with diabetes and/or obesity in a small study.

Lysosomal Storage Disorders are genetic conditions caused by enzyme deficiencies that lead to harmful substance buildup in cells, resulting in progressive organ damage. Common disorders in India ...

UAB is participating in a Phase I/II trial of AMT 191, a one time gene therapy designed to enable patients with classic Fabry disease to produce their own missing enzyme, potentially reducing the need ...

Ongoing clinical research at UNC could lead to a first-of-its-kind enzyme replacement therapy for Hunter syndrome, an ultra-rare disorder that causes progressive multisystem disease and neurologic ...

Children with Hunter Syndrome lack a certain enzyme in their blood. The Food and Drug Administration just approved an enzyme-replacement therapy for these patients. It had its start at UNC Hospitals.

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 ...

BioMarin Pharmaceutical is building up its pipeline with the acquisition of Inozyme Pharma in a $270 million deal centered on an enzyme replacement therapy that could become the first FDA-approved ...

If you or someone you know has Fabry disease, you may have heard of Galafold. This is the first and only medicine for Fabry disease that can be taken by mouth. Other treatments for Fabry disease, ...

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 ...

WALTHAM, Mass., June 24, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for ...