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

The regulator has received reports that a group of patients treated with Adzynma had neutralizing antibodies against the ...

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 ...

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.

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

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

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 ...

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 ...

Dublin, Oct. 02, 2025 (GLOBE NEWSWIRE) -- The "Mucopolysaccharidosis Market - A Global and Regional Analysis: Focus on Treatment, Disease Type, Route of Administration, End User, Country, and Region - ...

Now Ollie is making history as the world’s first successful gene therapy patient for this cruel condition. Ten months after ...

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

The first child in history has successfully been treated with a new genetic therapy for an ultra-rare developmental defect ...