We recently compiled a list of 11 Best Future Stocks to Buy Now. Denali Therapeutics Inc. stands sixth on our list among the ...

Scientists have successfully restored hearing in animal models of ENPP1 deficiency, a genetic disease in which individuals lack an enzyme essential for bone and blood vessel health. The rare disorder ...

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 ...

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

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.

Nevertheless, these data led to FDA approval in November 2023 for recombinant ADAMTS13 as prophylactic or on-demand enzyme replacement therapy for the extremely rare, chronic disorder that causes ...

Receipt of Fast Track designation underscores our belief that INZ-701 could serve as an important therapy for patients living with ABCC6 deficiency, notably for pediatric patients in whom this ...

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 ...

Pancreatic enzyme replacement therapy (PERT) is an oral treatment that replaces the enzymes the pancreas does not produce. In many people, symptoms will improve or resolve with PERT.

More information: Hemanth R. Nelvagal et al, Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep, Journal of Clinical Investigation (2022). DOI: 10.1172/JCI163107 ...

Ayla’s case, described Wednesday in the New England Journal of Medicine, was the first in which doctors provided this type of treatment — called enzyme replacement therapy, or ERT — in utero.