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Results for “Glycogen Storage Disease Type II (GSD-II)”

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Showing 20 of 0 results

Not applicableLooking for participantsNCT06557369
What this trial is testing

A Clinical Trial Evaluating the Safety and Efficacy of a New Light Combination Therapy Addressing Intermediate AMD

Who this might be right for
Intermediate AMD
Oculox Technologies SA 30
Testing effectiveness (Phase 2)Active Not RecruitingNCT05793307
What this trial is testing

Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug

Who this might be right for
Pompe Disease Infantile-Onset
GeneCradle Inc 16
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 applicableEnrolling By InvitationNCT06605612
What this trial is testing

Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders

Who this might be right for
Inclusion Body MyositisMyotonic DystrophyLimb-girdle and Facioscapulohumeral Muscular Dystrophies+9 more
LMU Klinikum 108
Post-approval studies (Phase 4)Active Not RecruitingNCT07652814
What this trial is testing

Safety and Efficacy of Avalglucosidase Alfa in Patients With Non-classic Pompe Disease Aged ≥ 5 Years

Who this might be right for
Glycogen Storage Disease Type II Late OnsetPompe Disease (Late-onset)Pompe Disease Late-Onset+2 more
Iris Plug 6
Not applicableStudy completedNCT03893240
What this trial is testing

Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease

Who this might be right for
Pompe DiseasePompe Disease (Late-onset)Glycogen Storage Disease Type 2+3 more
Spark Therapeutics, Inc. 61
Testing effectiveness (Phase 2)Active Not RecruitingNCT04174105
What this trial is testing

Gene Transfer Study in Patients With Late Onset Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)
Astellas Gene Therapies 11
Not applicableLooking for participantsNCT03564561
What this trial is testing

Natural History of Pompe Disease

Who this might be right for
Glycogen Storage Disease Type II, Adult
Assistance Publique - Hôpitaux de Paris 20
Post-approval studies (Phase 4)Study completedNCT00701129
What this trial is testing

An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type II
Genzyme, a Sanofi Company 4
Not applicableLooking for participantsNCT01665326
What this trial is testing

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

Who this might be right for
Pompe Disease
Duke University 400
Not applicableUnknownNCT02708784
What this trial is testing

An MRI Study on Muscular Diseases -Pompe Disease and Dystrophia Myotonica-

Who this might be right for
Glycogen Storage Disease Type 2Dystrophia Myotonica
University of Aarhus 60
Not applicableStudy completedNCT03285126
What this trial is testing

Pompe Gene Therapy- Screening for Eligibility

Who this might be right for
Pompe Disease (Late-onset)
Duke University 19
Not applicableApproved For MarketingNCT00520143
What this trial is testing

Alglucosidase Alfa Temporary Access Program

Who this might be right for
Glycogen Storage Disease Type II (GSD-II)Pompe Disease (Late-Onset)Acid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company
Testing effectiveness (Phase 2)Study completedNCT00765414
What this trial is testing

Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.

Who this might be right for
Pompe Disease Late-OnsetGlycogen Storage Disease Type II GSD II
Genzyme, a Sanofi Company 1
Not applicableStudy completedNCT04724213
What this trial is testing

Development of an Assessment Tool for Health-related Quality of Life in Children and Adolescents With Pompe Disease

Who this might be right for
Pompe Disease
University Children's Hospital, Zurich 101
Testing effectiveness (Phase 2)Study completedNCT00976352
What this trial is testing

Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease

Who this might be right for
Pompe Disease
University of Florida 9
Not applicableAvailableNCT03865836
What this trial is testing

Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease

Who this might be right for
Pompe Disease
Amicus Therapeutics
Not applicableLooking for participantsNCT07136844
What this trial is testing

Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology

Who this might be right for
Neuromuscular DiseasesObesity (Disorder)Myotonic Dystrophy 1+9 more
Centre Hospitalier Universitaire de Liege 300
Not applicableLooking for participantsNCT07834320
What this trial is testing

Pompe Disease RWE Long-term Monitoring Program

Who this might be right for
Pompe Disease
National Taiwan University Hospital 40
Testing effectiveness (Phase 2)Ended earlyNCT00688597
What this trial is testing

Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease

Who this might be right for
Pompe Disease
Amicus Therapeutics 3
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