Scientists have successfully restored hearing in animal models of ENPP1 deficiency, a genetic disease in which individuals ...

A study evaluating a pioneering lentivirus (LV)-mediated gene therapy trial for classical Fabry disease showed promising ...

Among patients with congenital thrombotic thrombocytopenic purpura, prophylactic therapy with recombinant ADAMTS13 restored normal levels of the enzyme, according to results of a randomized phase ...

Enzyme-replacement therapy may slow disease progression and improve motor function in children with Pompe disease, according to a poster from the 2024 Muscular Dystrophy Association Clinical ...

Primary Source New England Journal of Medicine Source Reference: Cohen J, et al "In utero enzyme-replacement therapy for infantile-onset Pompe's disease" N Engl J Med 2022; DOI: 10.1056/NEJMoa2200587.

Pombiliti and Opfolda together represent Amicus’s second commercialized therapy. The company’s first product, Galafold, treats Fabry disease, a different rare enzyme deficiency.

What would you do to save your child? This mom is fighting to fund a cure for her daughter's terminal illness.

BioMarin’s top product is Vimizim, approved in 2014 as an enzyme replacement therapy for mucopolysaccharidosis IVA. Vimizim generated $739.8 million in revenue last year.

Title: Somatic Outcomes in a Phase 1/2 Study of Weekly Intravenous DNL310 (Brain-Penetrant Enzyme Replacement Therapy) in Mucopolysaccharidosis Type II (Poster #43) Session: Clinical Applications ...

The global enzyme replacement therapy (ERT) targeting Pompe disease market is projected to attain a value of $350 million in 2025, growing at a compound annual growth rate (CAGR) of 12% between 2025 ...

A researcher says an experimental gene therapy for a rare inherited disorder is saving almost as much money for the treatment of five patients as the study itself costs.

Local News Enzyme Replacement Therapy Being Used To Treat Hunter Syndrome Posted 6:33 a.m. Nov 10, 2006 - Updated 5:11 a.m. Aug 23, 2006 ...