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Results for “Spinal Muscular Atrophy (SMA)”

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Showing 20 of 0 results

Not applicableNot Yet RecruitingNCT07530926
What this trial is testing

Adrenal& Bone Complications in Paediatric Patients Living With Spinal Muscular Atrophy (SMA)

Who this might be right for
SMA - Spinal Muscular Atrophy
Sheffield Children's NHS Foundation Trust 550
Early research (Phase 1)Study completedNCT02052791
What this trial is testing

An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246)

Who this might be right for
Spinal Muscular Atrophy
Biogen 47
Not applicableUnknownNCT00155168
What this trial is testing

Quantitative Analysis of SMN1 and SMN2 Gene Based on DHPLC System

Who this might be right for
Spinal Muscular Atrophy
National Taiwan University Hospital 500
Post-approval studies (Phase 4)Looking for participantsNCT05861986
What this trial is testing

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

Who this might be right for
Muscular Atrophy, Spinal
Hoffmann-La Roche 28
Testing effectiveness (Phase 2)Looking for participantsNCT05747261
What this trial is testing

Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy (BLUEBELL)

Who this might be right for
Spinal Muscular Atrophy (SMA)
Biocad 40
Not applicableEnrolling By InvitationNCT06772402
What this trial is testing

GCB-001 in Treatment of Patients With Type II (SMA) Spinal Muscular Atrophy

Who this might be right for
Spinal Muscular Atrophy Type 2
Genecombio Ltd. 6
Testing effectiveness (Phase 2)Study completedNCT02386553
What this trial is testing

Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Who this might be right for
Spinal Muscular Atrophy
Biogen 25
Not applicableStudy completedNCT02574390
What this trial is testing

Answer ALS: Individualized Initiative for ALS Discovery

Who this might be right for
Amyotrophic Lateral SclerosisPrimary Lateral SclerosisFlail Arm ALS+5 more
Johns Hopkins University 1,049
Large-scale testing (Phase 3)Study completedNCT01645787
What this trial is testing

Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients

Who this might be right for
Spinal Muscular Atrophy
Columbia University 11
Not applicableUnknownNCT05521217
What this trial is testing

Myotonometer Intra- and Inter Examiner Reliability on Spinal Muscular Atrophy

Who this might be right for
Muscle Tone PoorSpinal Muscular Atrophy
Istanbul Medipol University Hospital 30
Large-scale testing (Phase 3)Active Not RecruitingNCT05626855
What this trial is testing

Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab

Who this might be right for
Spinal Muscular AtrophySpinal Muscular Atrophy Type 3Spinal Muscular Atrophy Type 2+7 more
Scholar Rock, Inc. 238
Not applicableLooking for participantsNCT05789758
What this trial is testing

Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies

Who this might be right for
Muscular Atrophy, Spinal
Biogen 20
Testing effectiveness (Phase 2)Active Not RecruitingNCT03779334
What this trial is testing

Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Who this might be right for
Muscular Atrophy, Spinal
Hoffmann-La Roche 26
Testing effectiveness (Phase 2)Study completedNCT05517603
What this trial is testing

Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients

Who this might be right for
Spinal and Bulbar Muscular AtrophyKennedy's Disease
AnnJi Pharmaceutical Co., Ltd. 25
Early research (Phase 1)Ended earlyNCT05673109
What this trial is testing

AC176 for the Treatment of Metastatic Castration Resistant Prostate Cancer

Who this might be right for
Metastatic Castration Resistant Prostate Cancer
Accutar Biotechnology Inc 8
Not applicableUnknownNCT04825119
What this trial is testing

Hyperkinetic Movements in Patients With Disease of Motor Neurons and Their Response to Treatment With Nusinersen

Who this might be right for
TremorInvoluntary MovementsSMA+1 more
University Medical Centre Ljubljana 110
Not applicableStudy completedNCT02003937
What this trial is testing

Aerobic Training in Patients With Spinal Muscular Atrophy Type III

Who this might be right for
Spinal Muscular Atrophy
Rigshospitalet, Denmark 15
Not applicableLooking for participantsNCT04708431
What this trial is testing

Androgen Receptor, Implications for Health and Wellbeing: Natural History Study of Individuals With Androgen Insensitivity

Who this might be right for
Androgen Insensitivity SyndromeMetabolic Parameters in AIS, CAIS, PAIS and MAISTumor Formation in AIS, CAIS, PAIS and MAIS+1 more
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 650
Early research (Phase 1)Study completedNCT03988907
What this trial is testing

A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants

Who this might be right for
Spinal Muscular Atrophy
Hoffmann-La Roche 35
Not applicableStudy completedNCT01736553
What this trial is testing

Spinal Muscular Atrophy (SMA) Biomarkers Study in the Immediate Postnatal Period of Development

Who this might be right for
Spinal Muscular Atrophy (SMA)
Ohio State University 53
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