Intracerebral Gene Therapy for Sanfilippo Type A Syndrome

4 participants · First posted Nov 18, 2011

Biomarker for Sanfilippo Type A-B-C-D Disease (BioSanfilippo)

First posted Nov 24, 2014

Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

5 participants · First posted Sep 13, 2019

NAGLU gene AAV9 therapy (sanfilippo syndrome B)

gene therapy (adeno-associated virus, Sanfilippo syndrome)

Coenzyme A

C₂₁H₃₆N₇O₁₆P₃S · Rx

Abeona Therapeutics

United States

Sanfilippo Therapeutics SAS

A&A SMALL CHANGES HANDYPEOPLE

United States

Ultragenyx's UX111 Gene Therapy Shows Promise in Sanfilippo Syndrome Type A

FDA Approves Fayuvi, First-Ever Gene Therapy for Sanfilippo Syndrome Type A

Ultragenyx's UX111 Gene Therapy for Sanfilippo Syndrome Type A Receives FDA Priority Review