Researchers examined systemic delivery of a vector to replace the enzyme IDUA, which is deficient in patients with a rare enzyme deficiency disorder. The works describes how an injection of a vector ...

Most chemotherapy regimens rely on systemic administration of drugs leading to a wide array of toxicities. Using viral-vector-mediated gene modification of muscle tissues, we have developed a method ...

Early treatment is critical for mucopolysaccharidosis type I (MPS I), justifying its incorporation into newborn screening. Enzyme replacement therapy (ERT) treats MPS I, yet presumptions that ERT ...

Phase I and Pharmacokinetic Study of the Ribonucleotide Reductase Inhibitor, 3-Aminopyridine-2-Carboxaldehyde Thiosemicarbazone, Administered by 96-Hour Intravenous Continuous Infusion Virus-directed ...

Breaking News: Trump administration impacts continue to roil the life sciences sector Home » Autologous B-cell therapy WFX-001 corrects systemic α-GAL enzyme deficiency in vivo ...

FBX-101 clinical data demonstrating safety and initial efficacy from the RESKUE trial is being presented at the 2022 Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium ...

In patients naïve to HER2-targeted therapy, the objective response rate was 71% with a median duration of response of 9.2 months.

Acquisition is Strong Strategic Fit for BioMarin, Adding INZ-701, a Phase 3 Enzyme Replacement Therapy Being Developed for Treatment of ENPP1 Deficiency First Pivotal Data Readout in Children Expected ...

New Rochelle, NY, August 13, 2018--A new study has shown that a single injection of a novel adeno-associated vector (AAV)-based therapy can result in improved enzyme activ-ity and glycogen clearance ...

Please provide your email address to receive an email when new articles are posted on . NEW YORK — Systemically administered Janus kinase inhibitors hold potential for the treatment of alopecia areata ...

Phoenix-based Marlyn Nutraceuticals, dba Naturally Vitamins, has extended its contract with MUCOS Pharma of Germany giving them exclusive rights to distribute Wobenzym in the US. Wobenzym, which will ...

Walking Fish Therapeutics Inc. presented a new first-in-class autologous B-cell therapy, named WFX-001, being developed for the treatment of Fabry disease.