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PubPharm (373)
1
At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions : A Multi-Site Tissue Repository Providing Annotated Biospecimens for Approved Investigator-Directed Biomedical Research Initiatives
enthalten in:
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| 2024
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2
Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy : Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy
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| 2024
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3
Mechanisms and Treatment of Exercise Intolerance and Persistent Fatigue in Spinal Muscular Atrophy : Mechanisms and Treatment of Exercise Intolerance and Persistent Fatigue in Spinal Muscular Atrophy
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| 2024
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4
An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy : Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients With Later-Onset Spinal Muscular Atrophy (TOPAZ)
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| 2024
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5
High-intensity Interval Training in Patients With Spinal Muscular Atrophy : High-intensity Interval Training in Patients With Spinal Muscular Atrophy
enthalten in:
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| 2024
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6
A Placebo-controlled Study of Clenbuterol in Spinal and Bulbar Muscular Atrophy : A Placebo-controlled Study to Evaluate the Efficacy and Safety of Clenbuterol in Patients With Spinal and Bulbar Muscular Atrophy (SBMA)
enthalten in:
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| 2024
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7
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab-ONYX : An Open-Label, Multicenter, Extension Trial to Evaluate the Long-Term Safety and Efficacy of Apitegromab in Patients With Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab
enthalten in:
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| 2024
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8
A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy : A Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
enthalten in:
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| 2024
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9
A Review of the Management and Outcomes of Children With SMA in the West Midlands During 2017-2022 : A Review of the Management and Outcomes of Children With SMA in the West Midlands During 2017-2022
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| 2024
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10
Clinical, Molecular and Imaging Biomarkers in Spinal and Bulbar Muscular Atrophy (SBMA) : An Observational Study to Assess Clinical, Molecular and Imaging Biomarkers in Spinal and Bulbar Muscular Atrophy (SBMA)
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| 2024
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Thema: Atrophy
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Thema
Atrophy
345
Muscular Atrophy
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610
239
Study Type: Interventional
208
Muscular Atrophy, Spinal
159
Recruitment Status: Completed
96
Study Type: Observational
85
Recruitment Status: Recruiting
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Medical Condition: Spinal Muscular Atrophy
38
Phase: Phase 2
33
Spinal Muscular Atrophies of Childhood
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Recruitment Status: Active, not recruiting
24
Phase: Phase 3
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Spinal muscular atrophy
19
Recruitment Status: Not yet recruiting
17
Neuromuscular Diseases
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Phase: Phase 1
16
Recruitment Status: Unknown status
15
Recruitment Status: Terminated
15
Sarcopenia
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