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Results for “Mucopolysaccharidoses”

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Showing 20 of 0 results

Not applicableAvailableNCT05795361
What this trial is testing

Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

Who this might be right for
Hunter Syndrome
Takeda
Not applicableNot Yet RecruitingNCT07666269
What this trial is testing

Morphology in Oral Rare Syndromes & Artificial Intelligence for Clinical Diagnosis

Who this might be right for
Osteogenesis ImperfectaRare Bone DisordersHypophosphatemia+6 more
University Hospital, Bordeaux 240
Not applicableStudy completedNCT06064656
What this trial is testing

A Non-Interventional Study of Clinical Characteristics and Mortality of US Patients With Fibrodysplasia Ossificans Progressiva (FOP)

Who this might be right for
Fibrodysplasia Ossificans Progressiva (FOP)
Regeneron Pharmaceuticals 131
Not applicableUnknownNCT03227042
What this trial is testing

A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Who this might be right for
Mucopolysaccharidosis Type IIIB
Allievex Corporation 44
Large-scale testing (Phase 3)Enrolling By InvitationNCT06075537
What this trial is testing

An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

Who this might be right for
Mucopolysaccharidosis II
Denali Therapeutics Inc. 99
Large-scale testing (Phase 3)Active Not RecruitingNCT05208281
What this trial is testing

A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II

Who this might be right for
Mucopolysaccharidosis Type IIMetabolic Diseases
AO GENERIUM 32
Testing effectiveness (Phase 2)Ended earlyNCT01609062
What this trial is testing

Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome

Who this might be right for
Mucopolysaccharidosis IVAMorquio A SyndromeMPS IVA
BioMarin Pharmaceutical 25
Not applicableStudy completedNCT02936713
What this trial is testing

Effect of Controlled Diet Combined With a Fermented Milk Product on Gas-related Symptoms

Who this might be right for
Functional Gastrointestinal Disorders
Danone Global Research & Innovation Center 126
Testing effectiveness (Phase 2)Ended earlyNCT03315182
What this trial is testing

Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB

Who this might be right for
Mucopolysaccharidosis Type 3 B
Abeona Therapeutics, Inc 11
Not applicableAvailableNCT03775174
What this trial is testing

Expanded Access to Mepsevii

Who this might be right for
MPS VIIMucopolysaccharidosis VIISly Syndrome
Ultragenyx Pharmaceutical Inc
Large-scale testing (Phase 3)Active Not RecruitingNCT06031259
What this trial is testing

Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment

Who this might be right for
Hunter Syndrome
Takeda 6
Large-scale testing (Phase 3)Study completedNCT00146770
What this trial is testing

Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients

Who this might be right for
Mucopolysaccharidosis IHurler's SyndromeHurler-Scheie Syndrome+1 more
Genzyme, a Sanofi Company 45
Not applicableStudy completedNCT03292887
What this trial is testing

Hunter Outcome Survey (HOS)

Who this might be right for
Hunter Syndrome
Shire 1,443
Large-scale testing (Phase 3)Study completedNCT02432144
What this trial is testing

UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

Who this might be right for
Sly SyndromeMPS VIIMucopolysaccharidosis+1 more
Ultragenyx Pharmaceutical Inc 12
Testing effectiveness (Phase 2)Study completedNCT01043640
What this trial is testing

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Who this might be right for
MucopolysaccharidosisHurler SyndromeHunter Syndrome+10 more
Masonic Cancer Center, University of Minnesota 46
Testing effectiveness (Phase 2)Study completedNCT01155778
What this trial is testing

Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients

Who this might be right for
Mucopolysaccharidosis (MPS)
Shire 12
Not applicableStudy completedNCT03150069
What this trial is testing

Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?

Who this might be right for
Morquio DiseaseMPS - Mucopolysaccharidosis
Emory University 18
Not applicableNot Yet RecruitingNCT07712003
What this trial is testing

Natural History to Assess Disease in Patients With MPS IIIC

Who this might be right for
Sanfilippo Syndrome Type CMucopolysaccharidosis (MPS) IIIC
Phoenix Nest 35
Not applicableUnknownNCT06177847
What this trial is testing

Efficacy of BIS Monitoring in Deep Hypnotic State

Who this might be right for
Hypnotic; Withdrawal State With DeliriumNeurological DisorderPostoperative Delirium+4 more
Universitas Indonesia 50
Large-scale testing (Phase 3)Study completedNCT01645189
What this trial is testing

Safety and Efficacy of Hunterase

Who this might be right for
Hunter Syndrome
Green Cross Corporation 6
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