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Recruiting trial = Recruiting trial
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National clinical trial(s)

FRANCE

AUVERGNE-RHONE-ALPES
BRON

A randomized, placebo-controlled, double blind phase IIb study evaluating miglustat treatment in patients with Mucopolysaccharidosis type III (MPS III) or Sanfilippo syndrome - Terminated -
CHU de Lyon HCL - GH Est-Hôpital Femme Mère Enfant
Service d'endocrinologie et de diabétologie pédiatriques et maladies héréditaires du métabolisme

SPAIN

Galicia
SANTIAGO DE COMPOSTELA

Recruiting trial
Ongoing trial
Phase I/II Gene Therapy Clinical Trial of scAAV9.U1A.SGSH for Mucopolysaccharidosis IIIA - ES
Complejo Hospitalario Universitario de Santiago
Departamento de Pediatría