BioMarin Pharmaecutical has announced the enrollment of the first patient in a Phase 1/2 trial for BMN 250, as a treatment for Sanfilippo B Syndrome. “Children with Sanfilippo syndrome need therapies that target the root cause of this fatal genetic disease that currently has no approved treatment options,” said Jill Wood, Co-Founder and Treasurer, Jonah’s Just Begun Foundation to Cure Sanfilippo. “We applaud companies like BioMarin who are bringing scientific research into the clinic. This is a critical first step to finding potential treatments.”
For more information, visit the BioMarin website.