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.

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 ...

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, ...

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 ...

"There are three digestive enzymes made by your pancreas," says Mary Vouyiouklis Kellis, MD, a specialist in the department of endocrinology, diabetes and metabolism at the Cleveland Clinic. "Most ...

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 ...

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 ...

Long-Term Data Presented at the 22nd Annual WORLDSymposium™ Highlights Tralesinidase Alfa Enzyme Replacement Therapy’s Potential as the First Disease-Modifying Treatment Option for Sanfilippo Syndrome ...

(RTTNews) - uniQure N.V. (QURE), a gene therapy company developing treatments for severe genetic diseases, announced updated preliminary Phase 1/2a data for AMT-191, its investigational AAV gene ...