Analysis from continued follow-up of Phase 1/2 clinical trial data in Hunter syndrome (MPS II) reinforces potential for tividenofusp alfa (DNL310) to address full disease spectrum Launch readiness ...

The company had previously said that it expects to submit an application for approval of the therapy in the first quarter of ...

Pfizer is playing catch-up in the crowded field of obesity medicines, but it hoped to compete with a daily pill alternative to currently available injectable GLP-1 products. Instead, Pfizer is ...

BUSAN, South Korea--(BUSINESS WIRE)--NexThera Co., Ltd. (CEO SaeGwang Park) announced that it has submitted a Phase 1/2a Investigational New Drug (IND) application to the U.S. Food and Drug ...

Patients with early-onset lysosomal storage diseases are ideal candidates for prenatal therapy because organ damage starts in utero. We report the safety and efficacy results of in utero ...

Scientists headed by a team at the University of Tokyo have developed a new CRISPR-based gene-editing tool that they suggest could lead to better treatments for patients with genetic disorders. The ...

Fabry disease in mainland China is now mapped through the largest single-centre cohort to date, clarifying how α-galactosidase A deficiency, genotype and sex jointly sculpt the clinical spectrum.

Enzymes are biological catalysts that speed up chemical reactions in living organisms. They are essential for life, as they play a crucial role in various biological processes, such as digestion, ...

Polygenic scores aim to measure the cumulative effect of multiple genetic variants to make predictions about a person’s health. The most likely areas of potential use are in risk prediction, including ...