Juvenile idiopathic arthritis or JIA, the most frequent chronic inflammatory arthritis disorder affecting children, may co-occur with Fabry…
Articles by Marta Figueiredo, PhD
One year of treatment with PRX-102 (pegunigalsidase alfa), an investigational enzyme replacement therapy (ERT), was at least as effective as…
The U.S. Food and Drug Administration (FDA) has rejected Protalix BioTherapeutics and Chiesi Global Rare Diseases’ request for accelerated approval of PRX-102 (pegunigalsidase…
A Phase 1/2 clinical trial evaluating the investigational gene therapy 4D-310, developed by 4D Molecular Therapeutics (4DMT) for people…
NanoGLA — a potentially more effective version of enzyme replacement therapy that delivers the missing enzyme in Fabry disease…
A once-a-month regimen of the investigational enzyme replacement therapy (ERT) PRX-102 (pegunigalsidase alfa) is safe and effective at maintaining…
Freeline Therapeutics has developed a more robust test to detect the presence of neutralizing antibodies that affect the effectiveness…
One year of treatment with PRX-102 (pegunigalsidase alfa), an investigational enzyme replacement therapy (ERT), safely and effectively slowed kidney…
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of…
Using only an age-dependent threshold of estimated glomerular filtration rate (eGFR) — a marker of kidney function — indicating mild-to-moderate…