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

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.

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 ...

A three-year-old Oliver (Ollie) Chu has become the world’s first patient to receive a revolutionary stem cell gene therapy for Hunter syndrome.

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

Now Ollie is making history as the world’s first successful gene therapy patient for this cruel condition. Ten months after receiving revolutionary treatment at Royal Manchester Children’s Hospital, ...

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 - ...

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 ...

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 ...

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 ...

Xanthine oxidase inhibitors, primarily allopurinol and febuxostat, are the mainstay of treatment. The goal of chronic gout management includes lowering serum urate levels to below 6 mg/dL. 5 Overall, ...