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Results for “Hurler Disease”

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Enrolling by Invitation

Showing 10 of 0 results

Not applicableEnrolling By InvitationNCT06103487
What this trial is testing

Long Term Follow-Up for RGX-111

Who this might be right for
Mucopolysaccharidosis I
REGENXBIO Inc. 21
Not applicableLooking for participantsNCT07361536
What this trial is testing

Cardiac Structure and Function in MPS

Who this might be right for
MPS IMPS IV AMPS - Mucopolysaccharidosis+1 more
Children's Hospital of Orange County 240
Large-scale testing (Phase 3)Study completedNCT00912925
What this trial is testing

Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I

Who this might be right for
Mucopolysaccharidosis IHurlers SyndromeHurler-Scheie Syndrome
Genzyme, a Sanofi Company 45
Not applicableLooking for participantsNCT03333200
What this trial is testing

Longitudinal Study of Neurodegenerative Disorders

Who this might be right for
MLDKrabbe DiseaseALD+24 more
University of Pittsburgh 1,500
Not applicableEnded earlyNCT04628871
What this trial is testing

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

Who this might be right for
Hemophilia BMucopolysaccharidosis IMucopolysaccharidosis II
Sangamo Therapeutics 13
Early research (Phase 1)Ended earlyNCT00215527
What this trial is testing

Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I

Who this might be right for
Mucopolysaccharidosis ILysosomal Storage DiseasesSpinal Cord Compression
Patricia I. Dickson, M.D. 4
Not applicableStudy completedNCT05073783
What this trial is testing

Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting

Who this might be right for
Pompe DiseaseMucopolysaccharidosis Type I (MPS I)
Sanofi 57
Not applicableStudy completedNCT00144794
What this trial is testing

Mucopolysaccharidosis I (MPS I) Registry

Who this might be right for
Mucopolysaccharidosis I (MPS I)
Genzyme, a Sanofi Company 1,406
Testing effectiveness (Phase 2)Study completedNCT04453085
What this trial is testing

An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I)

Who this might be right for
Mucopolysaccharidosis I
JCR Pharmaceuticals Co., Ltd. 14
Early research (Phase 1)Study completedNCT00638547
What this trial is testing

Intrathecal Enzyme Replacement for Hurler Syndrome

Who this might be right for
Hurler Syndrome
Masonic Cancer Center, University of Minnesota 26