The devastating consequences of mucopolysaccharidosis II (MPS II), also called Hunter syndrome, may soon become preventable ...

Stem cells transplanted in utero (green) engrafted into fetal mouse brain tissue. Image credit: Q-H Nguyen/MacKenzie lab. Administering stem cell or enzyme therapy in utero may be a path to ...

Tividenofusp alfa treatment showed reduction and normalization in key disease biomarkers, stabilization or improvement in clinical endpoints ...

As the name implies, a rare disease is an illness that affects far fewer people than more common conditions, like breast cancer or heart disease. Today, however, 7,000 rare conditions have been ...

Full MIST2 dosing of intrapleural enzyme therapy varies widely across hospitals and is associated with a lower risk for ...

Please provide your email address to receive an email when new articles are posted on . Researchers analyzed 19 children with Pompe disease seen at a Texas-based children’s hospital.

Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window The first test ...

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 ...

FDA approves avalglucosidase alfa-ngpt (Nexviazyme) for the treatment of patients 1 year and older with late-onset Pompe disease. FDA announced yesterday the approval of avalglucosidase alfa-ngpt, ...

A new study led by University of Pittsburgh and UPMC Hillman Cancer Center researchers shows that an enzyme called PARP1 is involved in repair of telomeres, the lengths of DNA that protect the tips of ...

Please provide your email address to receive an email when new articles are posted on . A phase 3 trial evaluating intrapleural enzyme therapy vs. video-assisted thoracoscopic surgery is feasible.

Obesity is a global epidemic and a major cause of morbidity and mortality because it increases the risk for comorbidities, including heart disease and fatty liver disease (MASLD).