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 ...

Relationship Between Participation in ASCO's Quality Oncology Practice Initiative Program and American Board of Internal Medicine's Maintenance of Certification Program We sought to characterize ...

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 ...

While lobectomy is the surgical gold standard for early-stage NSCLC, some patients will continue to need pneumonectomy to achieve radical resection. Historically, the use of adjuvant and neoadjuvant ...

Any practitioner who cares for patients with psoriasis should be familiar with and make use of systemic therapies. These medications are clearly indicated for patients who have diffuse disease where ...

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 ...

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.