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77 studies found for:    Congenital Myopathy
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Rank Status Study
21 Recruiting Clinical Study on the Safety of CNT-02 for TGCV and NLSD-M
Conditions: Primary Triglyceride Deposit Cardiomyovasculopathy (TGCV);   Neutral Lipid Storage Disease With Myopathy (NLSD-M)
Intervention: Dietary Supplement: CNT-02
22 Enrolling by invitation Efficacy of Sialic Acid GNE Related Thrombocytopenia
Condition: Thrombocytopenia
Intervention: Drug: Sialic Acid-Extended Release
23 Completed The Effect of Fibrate Therapy in Two Patients With Neutral Lipid Storage Disease With Myopathy (NLSDM)
Condition: Neutral Lipid Storage Disease
Intervention: Drug: Fibrate treatment
24 Completed MRI and Muscle Involvement in Patients With Mutations in GMPPB
Condition: Limb-girdle Muscular Dystrophy
Intervention:
25 Enrolling by invitation Treatment of TNNT1-Myopathy With L-Tyrosine.
Condition: Nemaline Myopathy
Interventions: Drug: L-Tyrosine;   Drug: Placebo
26 Active, not recruiting The Natural History of Congenital Trigger Thumbs
Conditions: Congenital Trigger Thumb;   Tenosynovitis
Intervention:
27 Recruiting Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO)
Condition: Congenital Muscular Dystrophy
Intervention: Drug: Omigapil
28 Completed A Study to Test Lung Stretch Therapy (Hyperinsufflation) in Children With Collagen VI Muscular Dystrophy
Condition: Congenital Muscular Dystrophy
Intervention: Other: Hyperinsufflation therapy
29 Recruiting Myotonic Dystrophy Family Registry
Conditions: Myotonic Dystrophy;   Congenital Myotonic Dystrophy;   Myotonic Dystrophy 1;   Myotonic Dystrophy 2;   Dystrophia Myotonica;   Dystrophia Myotonica 1;   Dystrophia Myotonica 2;   Myotonia Dystrophica;   Myotonic Dystrophy, Congenital;   Myotonic Myopathy, Proximal;   PROMM (Proximal Myotonic Myopathy);   Proximal Myotonic Myopathy;   Steinert Disease;   Steinert Myotonic Dystrophy;   Steinert's Disease;   Myotonia Atrophica
Intervention: Other: Patient-entered data
30 Recruiting The Savella Pregnancy Registry
Condition: Fibromyalgia
Intervention:
31 Recruiting Calibration and Validation of the PROMIS and Neuro-QOL Questionnaires in Cerebral Palsy and Congenital Muscular Dystrophy
Conditions: Cerebral Palsy;   Neuromuscular Disease
Intervention:
32 Available 3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenia (CM)
Conditions: Lambert-Eaton Myasthenic Syndrome (LEMS);   Congenital Myasthenia (CM)
Intervention: Drug: 3,4-diaminopyridine
33 Recruiting Genetic and Physical Study of Childhood Nerve and Muscle Disorders
Conditions: Muscular Dystrophies;   Muscle Myopathies;   Hereditary Spastic Paraplegias;   Inherited Neuropathies;   Inherited Neuromuscular Conditions
Intervention:
34 Recruiting Study of Ranolazine in Myotonia Congenita and Paramyotonia Congenita
Conditions: Myotonia Congenita (Enrollment Complete);   Paramyotonia Congenita
Intervention: Drug: Ranolazine
35 Recruiting Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy
Condition: Myotonic Dystrophy 1
Intervention: Drug: Tideglusib
36 Completed Relations Between Myotonia and Fitness
Condition: Congenital Myotonia
Intervention: Other: Training
37 Terminated European Home Mechanical Ventilation Registry
Conditions: Pulmonary Disease, Chronic Obstructive;   Amyotrophic Lateral Sclerosis;   Spinal Cord Injury;   Muscular Dystrophies;   Obesity Hypoventilation Syndrome;   Kyphoscoliosis;   Congenital Central Hypoventilation Syndrome;   Duchenne Muscular Dystrophy;   Myopathies;   Myotonic Dystrophy
Intervention:
38 Recruiting Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry
Conditions: Myotonic Dystrophy;   Facioscapulohumeral Muscular Dystrophy;   Muscular Dystrophy;   Myotonic Dystrophy Type 1;   Myotonic Dystrophy Type 2;   Congenital Myotonic Dystrophy;   PROMM (Proximal Myotonic Myopathy);   Steinert's Disease;   Myotonic Muscular Dystrophy
Intervention:
39 Completed Mexiletine and Non Dystrophic Myotonias
Conditions: Non-dystrophic Myotonias;   Paramyotonia Congenita;   Myotonia Congenita
Interventions: Drug: Mexiletine;   Drug: placebo
40 Recruiting International Registry Study of Neutral Lipid Storage Disease (NLSD) / Triglyceride Deposit Cardiomyovasculopathy (TGCV) and Related Diseases
Condition: Neutral Lipid Storage Disease
Intervention:

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Study has passed its completion date and status has not been verified in more than two years.