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PubPharm (1.629)
1
Study of Inherited Neurological Disorders : Clinical and Molecular Manifestations of Inherited Neurological Disorders
enthalten in:
ClinicalTrials.gov
| 2024
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2
Genetic and Physical Study of Childhood Nerve and Muscle Disorders : Clinical and Molecular Manifestations of Neuromuscular and Neurogenetic Disorders of Childhood
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| 2024
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3
A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment : A Phase 2, Open-Label, Multiple Ascending Dose Study of PGN-EDO51 With a Long-Term Extension in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (CONNECT1-EDO51)
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| 2024
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4
A Study to Evaluate the Safety and Tolerability of GEN6050X in Duchenne Muscular Dystrophy. : A Single-arm, Open-label, Single-center Study to Evaluate the Safety and Tolerability of Intravenous GEN6050X Gene Therapy in Ambulatory Boys With Duchenne Muscular Dystrophy (DMD).
enthalten in:
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| 2024
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5
Evolution of the Functional and Muscular State of Patients With Muscular Dystrophy 2A Belts : Evolution of the Functional and Muscular State and Quality of Life of Patients With Limb-girdle Muscular Dystrophy 2A: CALNATHIS Prospective Cohort Study
enthalten in:
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| 2024
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6
Skeletal Muscle Biomarkers in People With Fragile Sarcolemmal Muscular Dystrophy : Pilot Study to Assess Biomarkers of Changes in Barrier Function of Skeletal Muscle in Patients With a Fragile Sarcolemmal Muscular Dystrophy
enthalten in:
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| 2024
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7
Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I) : A Phase 3 Randomized, Placebo-controlled, Double-blind Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)
enthalten in:
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| 2024
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8
Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping : A Phase 1/2, Randomized, Placebo-controlled, Double-blind, Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effects of Single and Multiple Ascending Doses of AOC 1044 Administered Intravenously to Healthy Adult Volunteers and Participants With DMD Mutations Amenable to Exon 44 Skipping
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| 2024
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9
Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study : Gait Analysis Parameter, Stair Climbing and Upper Limb Evaluation in Patients With Muscular Pathology and in Control Subjects: The ActiLiège Next Study
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| 2024
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10
Upper Extremity Muscle Strength, Balance and Functional Skills in DMD : The Association Between Upper Extremity Muscle Strength, Balance and Functional Skills in Children With Duchenne Muscular Dystrophy
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| 2024
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Study Type: Interventional
597
Muscular Dystrophies
548
Recruitment Status: Not yet recruiting
428
Phase: Phase 3
399
Muscular Dystrophy, Duchenne
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Recruitment Status: Completed
266
Study Type: Observational
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Phase: Phase 2
251
Recruitment Status: Recruiting
194
Recruitment Status: Authorised-recruitment may ...
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Phase: Phase 1
134
Medical Condition: Duchenne Muscular Dystrophy
88
Medical Condition: Duchenne Muscular Dystrophy ...
62
Medical Condition: Duchenne muscular dystrophy
60
Muscular Dystrophy, Facioscapulohumeral
52
Muscular Dystrophies, Limb-Girdle
52
Recruitment Status: Active, not recruiting
51
Recruitment Status: Terminated
50
Recruitment Status: Complete: follow-up complete
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