SANFILIPPO MUCOPOLYSACCHARIDOSES

Clinical trials for SANFILIPPO MUCOPOLYSACCHARIDOSES explained in plain language.

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New hope for kids with devastating brain diseases: experimental cell therapy aims to halt progression

Disease control Recruiting now

This early-stage study is testing the safety of adding a special type of cell, derived from umbilical cord blood, to a standard cord blood transplant for children with rare inherited metabolic diseases that damage the brain. The goal is to see if delivering these cells directly i…

Matched conditions: SANFILIPPO MUCOPOLYSACCHARIDOSES

Phase: PHASE1 • Sponsor: Joanne Kurtzberg, MD • Aim: Disease control

Last updated Apr 01, 2026 14:42 UTC

SANFILIPPO MUCOPOLYSACCHARIDOSES

Get alerted when new SANFILIPPO MUCOPOLYSACCHARIDOSES trials appear

New hope for kids with devastating brain diseases: experimental cell therapy aims to halt progression