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Results for “Rare Genetic Muscle Diseases”

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Showing 11 of 0 results

Early research (Phase 1)Active Not RecruitingNCT03478462
What this trial is testing

Dose Escalation Study of CLR 131 in Pediatric Relapsed/Refractory Malignant Tumors Including Neuroblastoma and Sarcomas

Who this might be right for
Pediatric Solid TumorPediatric LymphomaPediatric Brain Tumor+5 more
Cellectar Biosciences, Inc. 30
Not applicableActive Not RecruitingNCT06809049
What this trial is testing

Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1)

Who this might be right for
Myotonic Dystrophy, CongenitalMyotonic Dystrophy, Type 1 (DM1)Myotonic Dystrophy Type 1+1 more
Hanns Lochmuller 13
Early research (Phase 1)Not Yet RecruitingNCT07787650
What this trial is testing

S-606001 as Monotherapy in Participants With Late-onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease
Shionogi 15
Not applicableStudy completedNCT00004770
What this trial is testing

Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy

Who this might be right for
Mitochondrial Myopathy
National Center for Research Resources (NCRR) 1
Not applicableStudy completedNCT00004353
What this trial is testing

Study of the Metabolism of Pyruvate and Related Problems in Patients With Lactic Acidemia

Who this might be right for
Mitochondrial MyopathyMELAS SyndromeLactic Acidosis
National Center for Research Resources (NCRR) 35
Not applicableLooking for participantsNCT06795152
What this trial is testing

Rare Glycogen Storage Diseases Natural History Study

Who this might be right for
Glycogen Storage DiseaseGSD Type 0AGSD Type 0B+12 more
Duke University 200
Not applicableStudy completedNCT00004685
What this trial is testing

Randomized Study of Albuterol in Patients With Facioscapulohumeral Muscular Dystrophy

Who this might be right for
Muscular Dystrophy, Facioscapulohumeral
Ohio State University 90
Not applicableUnknownNCT05092230
What this trial is testing

Frequency of Pompe Disease in Patients With Myalgia With or Without Hyper Ckemia - Data From the Reference Center (CERCA)

Who this might be right for
Pompe Disease
University Hospital Center of Martinique 100
Large-scale testing (Phase 3)Study completedNCT00004802
What this trial is testing

Phase III Randomized, Double-Blind, Placebo-Controlled Study of Dichlorphenamide for Periodic Paralyses and Associated Sodium Channel Disorders

Who this might be right for
Paralysis, Hyperkalemic PeriodicHypokalemic Periodic ParalysisParamyotonia Congenita
National Center for Research Resources (NCRR) 64
Not applicableStudy completedNCT03680365
What this trial is testing

Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families

Who this might be right for
Duchenne Muscular DystrophyBurden, DependencyDisability Physical+3 more
Jett Foundation, Inc. 60
Not applicableLooking for participantsNCT07127978
What this trial is testing

A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy

Who this might be right for
Duchene Muscular Dystrophy
ITF Therapeutics LLC 300