Purpose: Enzyme replacement therapy (ERT) is a promising therapeutic intervention for lysosomal storage diseases. Posttranslationally engineered human β ...

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 ...

FDA reports rare neutralizing antibodies, including a pediatric death, in patients treated with Takeda's Adzynma for congenital TTP.

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 ...

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.

In the United States about 5%-8% of the population is affected by an autoimmune disorder. Other reports, however, estimate a higher prevalence of various types of autoimmune disorders due to complex ...

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, ...

Nobel laureate David Baker’s latest foundation model can design proteins that interact to any biomolecule for broad biotech applications.

A California toddler is the first person in the world to receive gene therapy to treat his devastating disease.

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.