New review highlights evolving Pompe disease management, from screening to next-generation therapies and monitoring. Read ...

Protalix Biotherapeutics Inc (NYSE-A:PLX, FRA:PBDA) announced that the European Commission has approved a new dosing regimen ...

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 ...

This press release is intended for US audiences for transparency relative to global news for the Fabry community. This dosing regimen for ...

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 ...

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 ...

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.

Executives from Spruce Biosciences (NASDAQ:SPRB) outlined regulatory plans, clinical data highlights, and commercial considerations for the company’s lead program in Sanfilippo syndrome type B (MPS ...

In early trial data, INZ-701 was associated with several clinical benefits including a reduction or stabilization of carotid intima-media thickness. The Food and Drug Administration (FDA) has granted ...