Enzyme replacement therapy offers hope for ultra-rare Hunter syndrome

Ongoing clinical research at UNC could lead to a first-of-its-kind enzyme replacement therapy for Hunter syndrome, an ...
WebMD

Understanding Galafold: How This Oral Treatment Works for Fabry Disease

If you or someone you know has Fabry disease, you may have heard of Galafold. This is the first and only medicine for Fabry disease that can be taken by mouth. Other treatments for Fabry disease, ...
Healio

Enzyme replacement therapy curbs bleeding events in congenital blood clotting disorder

Please provide your email address to receive an email when new articles are posted on . Mean maximum ADAMTS13 activity after recombinant ADAMTS13 exceeded 100%. Markedly fewer patients receiving ...
Healio

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 ...
MedPage Today

Impact of Recombinant Replacement Therapy Detailed in Genetic Clotting Disorder

A recombinant replacement for the ADAMTS13 protein (Adzynma) missing in congenital thrombotic thrombocytopenic purpura (TTP) restored levels and reduced acute TTP events, according to interim trial ...
Medical Xpress

Therapeutic or Preventive Procedure:

A study evaluating a pioneering lentivirus (LV)-mediated gene therapy trial for classical Fabry disease showed promising results over five years, indicating a potential breakthrough in treatment for ...