Enzyme Replacement Therapy
Food allergy less common among children with cystic fibrosis
SAN DIEGO — In a cohort of nearly 300 pediatric patients with cystic fibrosis, food allergy was found in less than 4% of the population, which was a lower proportion than the general pediatric population, according to a presentation here.
Addressing gaps in standard of care may improve treatment for adults with Fabry disease
Addressing disparities in standard of care in disease monitoring and perception of disease progression could improve treatment for adults with Fabry disease, according to published data.
Enzyme-replacement therapy improves outcomes in Pompe disease
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 & Scientific Conference.
Study: Pegunigalsidase alfa noninferior to agalsidase beta in slowing eGFR decline
Pegunigalsidase alfa was noninferior to agalsidase beta in slowing the rate of eGFR decline during a 2-year period and had lower rates of adverse events and infusion reactions, according to recently published data.
FDA approves Adzynma as first treatment for blood clotting disorder
The FDA approved the enzyme replacement therapy Adzynma for the prophylactic or on-demand treatment of adults and pediatric patients with congenital thrombotic thrombocytopenic purpura, a rare blood clotting disorder.
FDA approves Lamzede for non-CNS alpha-mannosidosis in adults, children
The FDA has approved Lamzede, an enzyme replacement therapy for non-central nervous system manifestations of alpha-mannosidosis in adult and pediatric patients, Chiesi Global Rare Diseases announced in a press release.