Get expert advice on navigating your LOPD journey, from starting ERT treatments and building a specialist care team to ...

Please provide your email address to receive an email when new articles are posted on . Researchers analyzed 19 children with Pompe disease seen at a Texas-based children’s hospital.

Orsini, a leader in rare disease pharmacy solutions, announced today that it is the exclusive specialty pharmacy partner for Denali Therapeutics' AVLAYAH™ (tividenofusp alfa-eknm), an FDA-approved ...

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 ...

Orsini, a provider of rare disease pharmacy solutions, is the exclusive specialty pharmacy partner for Denali Therapeutics' AVLAYAH, an FDA-approved enzyme replacement therapy ...

Ongoing clinical research at UNC could lead to a first-of-its-kind enzyme replacement therapy for Hunter syndrome, an ultra-rare disorder that causes progressive multisystem disease and neurologic ...

Long-Term Data Presented at the 22nd Annual WORLDSymposium™ Highlights Tralesinidase Alfa Enzyme Replacement Therapy’s Potential as the First Disease-Modifying Treatment Option for Sanfilippo Syndrome ...

Enzyme replacement therapy (ERT), which provides a synthetic version of missing GAA enzyme, is the primary treatment for this condition. It’s not a cure, but it can address symptoms for people in all ...

Breakthrough Therapy Designation Supported by Integrated Long-Term Clinical Data Demonstrating Normalization in Cerebral Spinal Fluid Heparan Sulfate Non-Reducing End (CSF HS-NRE) U.S. FDA Confirmed ...

An investigational gene therapy showed sustained clinical efficacy for children born with ADA-SCID. In a study of 62 children, overall survival after treatment was 100% and event-free survival was 95% ...