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Results for “Kugelberg-Welander Disease”

32 trials

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Showing 20 of 32 results

Not applicableUnknownNCT05272969
What this trial is testing

Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)Inclusion Body MyositisSpinal Muscular Atrophy Type 3+1 more
LMU Klinikum 95
Not applicableStudy completedNCT04907162
What this trial is testing

Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders

Who this might be right for
Pompe Disease (Late-onset)Myotonic Dystrophy Type 1 (DM1)Myotonic Dystrophy Type 2+4 more
LMU Klinikum 82
Testing effectiveness (Phase 2)Study completedNCT00568802
What this trial is testing

A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients

Who this might be right for
Muscular Atrophy, Spinal
Stanford University 27
Testing effectiveness (Phase 2)Looking for participantsNCT06421831
What this trial is testing

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients

Who this might be right for
Spinal Muscular Atrophy Type 3
GeneCradle Inc 21
Testing effectiveness (Phase 2)Ended earlyNCT03819660
What this trial is testing

Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3

Who this might be right for
Spinal Muscular Atrophy Type 3
Catalyst Pharmaceuticals, Inc. 13
Large-scale testing (Phase 3)Study completedNCT05156320
What this trial is testing

Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

Who this might be right for
Spinal Muscular AtrophySpinal Muscular Atrophy Type 3Spinal Muscular Atrophy Type 2+7 more
Scholar Rock, Inc. 188
Not applicableStudy completedNCT00756821
What this trial is testing

A Pilot Study of Biomarkers for Spinal Muscular Atrophy

Who this might be right for
Spinal Muscular Atrophy
Carelon Research 130
Not applicableLooking for participantsNCT07400198
What this trial is testing

Gait and Bone Health in SMA

Who this might be right for
Spinal Muscular Atrophy Type 3Ambulatory Spinal Muscular Atrophy
Jacqueline Montes 22
Testing effectiveness (Phase 2)Study completedNCT03921528
What this trial is testing

An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy

Who this might be right for
Spinal Muscular AtrophySpinal Muscular Atrophy Type 3Spinal Muscular Atrophy Type 2+6 more
Scholar Rock, Inc. 58
Testing effectiveness (Phase 2)Looking for participantsNCT07047144
What this trial is testing

Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

Who this might be right for
Spinal Muscular AtrophySMASpinal Muscular Atrophy Type 2+3 more
Scholar Rock, Inc. 52
Not applicableActive Not RecruitingNCT06300996
What this trial is testing

Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb

Who this might be right for
Spinal Muscular AtrophySpinal Muscular Atrophy Type 3Spinal Muscular Atrophy Type II+2 more
Marco Capogrosso 3
Not applicableUnknownNCT06137612
What this trial is testing

Spinal Cord Gray Matter Imaging in Spinal Muscular Atrophy

Who this might be right for
Spinal Muscular Atrophy Type IIISpinal Muscular Atrophy Type II
University Hospital, Basel, Switzerland 52
Not applicableUnknownNCT04690998
What this trial is testing

Outcome Measures and Biomarkers in a Cohort of Spinal Muscular Atrophy Type III/ IV Patients

Who this might be right for
Spinal Muscular Atrophy
Assistance Publique Hopitaux De Marseille 104
Not applicableStudy completedNCT04193085
What this trial is testing

Wearable Technology to Assess Gait Function in SMA and DMD

Who this might be right for
Spinal Muscular Atrophy Type 3Duchenne Muscular Dystrophy
Columbia University 39
Not applicableStudy completedNCT00466349
What this trial is testing

International SMA Patient Registry

Who this might be right for
Muscular Atrophy, Spinal
Indiana University 3,000
Not applicableLooking for participantsNCT06562283
What this trial is testing

Evaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular Atrophy

Who this might be right for
Spinal AmyotrophyInfantile Spinal Muscular AtrophyJuvenile Spinal Muscular Atrophy
Centre Hospitalier Universitaire de Saint Etienne 80
Not applicableEnded earlyNCT03056144
What this trial is testing

Whole Body Vibration Therapy in Children With Spinal Muscular Atrophy

Who this might be right for
Spinal Muscular Atrophy Type 3
The Hong Kong Polytechnic University 1
Not applicableStudy completedNCT04404764
What this trial is testing

Characterization of the Clinical-epidemiological Profile of Patients With SMA5q Types II and III: Observational Study

Who this might be right for
Spinal Muscular Atrophy
Hospital Israelita Albert Einstein 155
Not applicableLooking for participantsNCT06839469
What this trial is testing

Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders

Who this might be right for
Spinal Muscular Atrophy Type 3Duchenne Muscular Dystrophy (DMD)
Columbia University 106
Large-scale testing (Phase 3)Active Not RecruitingNCT04042025
What this trial is testing

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

Who this might be right for
Spinal Muscular Atrophy Type ISpinal Muscular Atrophy Type IISpinal Muscular Atrophy Type III+1 more
Novartis Gene Therapies 85
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