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Conditions: Muscular Atrophy, Spinal
Interventions: Placebo, Risdiplam; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 2 Years to 25 Years
Enrollment: 231 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2023
U.S. locations: 2
States / cities: Palo Alto, California • New York, New York

Conditions: Spinal Muscular Atrophy (SMA), Carrier Screening, Genetic Testing
Interventions: Blood draw; Procedure
Lead sponsor: Natera, Inc.; Industry
Eligibility: 18 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2012
U.S. locations: 1
States / cities: San Carlos, California

Conditions: Amyotrophic Lateral Sclerosis, Progressive Muscular Atrophy
Interventions: Not listed
Lead sponsor: Duke University; Other
Eligibility: Not listed
Enrollment: 26 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2023
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Prostate Cancer
Interventions: Nilutamide, Dasatinib; Drug
Lead sponsor: Duke University; Other
Eligibility: 18 Years and older · Male only
Enrollment: 57 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2013
U.S. locations: 3
States / cities: Durham, North Carolina • Portland, Oregon • Seattle, Washington

Conditions: Muscular Atrophy, Spinal
Interventions: Risdiplam; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 1 Month to 7 Months
Enrollment: 62 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2023
U.S. locations: 4
States / cities: Palo Alto, California • Chicago, Illinois • Boston, Massachusetts + 1 more

Conditions: Spinal Muscular Atrophy
Interventions: Not listed
Lead sponsor: Massachusetts General Hospital; Other
Eligibility: Not listed
Enrollment: 1,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: Started 2016
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Spinal Muscular Atrophy Type 3, Ambulatory Spinal Muscular Atrophy
Interventions: Not listed
Lead sponsor: Jacqueline Montes; Other
Eligibility: 8 Years to 50 Years
Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2027
U.S. locations: 1
States / cities: New York, New York

Conditions: Muscular Atrophy, Spinal
Interventions: Nusinersen; Drug
Lead sponsor: Biogen; Industry
Eligibility: 2 Years and older
Enrollment: 115 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2026
U.S. locations: 7
States / cities: Sacramento, California • Aurora, Colorado • Chicago, Illinois + 4 more

Conditions: Advanced Lung Disease, Lung Transplant, Extracorporeal Membrane Oxygenation
Interventions: MRP and NMES(neuromuscular electric stimulation), Standard of Care; Device · Other
Lead sponsor: University of Maryland, Baltimore; Other
Eligibility: 18 Years to 80 Years
Enrollment: 70 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2027
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Neurologic Disorder, Paralysis, Paralysis; Stroke, Amyotrophic Lateral Sclerosis, Muscular Dystrophies, Spinal Cord Injuries, Stroke, Lacunar, Stroke Brainstem, Cervical Spinal Cord Injury, Spinal Muscular Atrophy, Tetraplegic; Paralysis, Quadriplegia/Tetraplegia
Interventions: Motor Neuroprosthesis (MNP); Device
Lead sponsor: Synchron Medical, Inc.; Industry
Eligibility: 21 Years to 75 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2026
U.S. locations: 3
States / cities: Buffalo, New York • New York, New York • Pittsburgh, Pennsylvania

Conditions: Spinal Muscular Atrophy
Interventions: Not listed
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: Up to 5 Years
Enrollment: 162 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022
U.S. locations: 1
States / cities: Bannockburn, Illinois

Conditions: Muscular Atrophy, Spinal
Interventions: Hydroxyurea, Placebo to match hydroxyurea; Drug
Lead sponsor: Stanford University; Other
Eligibility: Up to 2 Years
Enrollment: 29 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2012
U.S. locations: 1
States / cities: Stanford, California

Conditions: Muscular Atrophy, Spinal
Interventions: Salanersen, Sham Procedure; Drug · Procedure
Lead sponsor: Biogen; Industry
Eligibility: 0 Days to 7 Months
Enrollment: 42 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2033
U.S. locations: 1
States / cities: Flower Mound, Texas

Conditions: Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome, Bassen-Kornzweig Syndrome, Batten Disease, Best Disease, Choroidal Dystrophy, Choroideremia, Cone Dystrophy, Cone-Rod Dystrophy, Congenital Stationary Night Blindness, Enhanced S-Cone Syndrome, Fundus Albipunctatus, Goldmann-Favre Syndrome, Gyrate Atrophy, Juvenile Macular Degeneration, Kearns-Sayre Syndrome, Leber Congenital Amaurosis, Refsum Syndrome, Retinitis Pigmentosa, Retinitis Punctata Albescens, Retinoschisis, Rod-Cone Dystrophy, Rod Dystrophy, Rod Monochromacy, Stargardt Disease, Usher Syndrome
Interventions: Not listed
Lead sponsor: Foundation Fighting Blindness; Other
Eligibility: Not listed
Enrollment: 20,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2014 – 2037
U.S. locations: 1
States / cities: Columbia, Maryland

Conditions: Spinal Muscular Atrophy, Charcot-Marie-Tooth Disease, Muscular Dystrophy, Spinal Muscular Atrophy With Respiratory Distress 1, Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Neuromuscular Disease, Peroneal Muscular Atrophy
Interventions: sample collection; Other
Lead sponsor: Nemours Children's Clinic; Other
Eligibility: 1 Month and older
Enrollment: 230 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2015 – 2024
U.S. locations: 3
States / cities: Wilmington, Delaware • Jacksonville, Florida • Orlando, Florida

Conditions: Amyotrophic Lateral Sclerosis, Progressive Muscular Atrophy, Motor Neuron Disease
Interventions: Not listed
Lead sponsor: Duke University; Other
Eligibility: Not listed
Enrollment: 25 participants
Timeline: 2018 – 2020
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Type 2 Spinal Muscular Atrophy
Interventions: OAV101, Sham control; Genetic · Procedure
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 2 Years to 17 Years
Enrollment: 126 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 6
States / cities: Farmington, Connecticut • Chicago, Illinois • Strasburg, Pennsylvania + 2 more

Conditions: Spinal Muscular Atrophy
Interventions: nusinersen, Sham procedure; Drug · Procedure
Lead sponsor: Biogen; Industry
Eligibility: Up to 210 Days
Enrollment: 122 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2016
U.S. locations: 12
States / cities: Los Angeles, California • Aurora, Colorado • Orlando, Florida + 9 more

Conditions: Mitochondrial Disorders, Mitochondrial Genetic Disorders, Mitochondrial Diseases, Disorder of Mitochondrial Respiratory Chain Complexes, Deletion and Duplication of Mitochondrial DNA
Interventions: Not listed
Lead sponsor: Columbia University; Other
Eligibility: Not listed
Enrollment: 1,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2011 – 2026
U.S. locations: 16
States / cities: San Diego, California • Stanford, California • Aurora, Colorado + 12 more

Conditions: Spinal Muscular Atrophy (SMA)
Interventions: ARGX-119 IV, Placebo IV; Biological · Other
Lead sponsor: argenx; Industry
Eligibility: 5 Years to 17 Years
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2029
U.S. locations: 16
States / cities: Little Rock, Arkansas • San Diego, California • Stanford, California + 13 more

Conditions: Spinal Muscular Atrophy
Interventions: Not listed
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: 5 Years to 20 Years
Enrollment: 24 participants
Timeline: 2025 – 2028
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: Motor Neuron Disease
Interventions: FLX-787-ODT (orally disintegrating tablet), Placebo ODT; Drug
Lead sponsor: Flex Pharma, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 54 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2018
U.S. locations: 35
States / cities: Scottsdale, Arizona • Sacramento, California • San Francisco, California + 29 more

Conditions: Motor Neuron Disease
Interventions: A functional exercise program, A stretching exercise program; Behavioral
Lead sponsor: National Institute of Neurological Disorders and Stroke (NINDS); NIH
Eligibility: 18 Years to 90 Years · Male only
Enrollment: 61 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2018
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Metastatic Castration Resistant Prostate Cancer
Interventions: Rinzimetostat, Darolutamide, Enzalutamide, Docetaxel; Drug
Lead sponsor: ORIC Pharmaceuticals; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 600 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2030
U.S. locations: 7
States / cities: Jeffersonville, Indiana • Charlotte, North Carolina • Canton, Ohio + 4 more

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A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants

Multi-disease Carrier Screening Test Validation

Study of ALS Reversals 2: Genetic Analyses

Genomic Guided Therapy With Dasatinib or Nilutamide in Metastatic Castration-Resistant Prostate Cancer

Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy

Prospective Evaluation of Infants With Spinal Muscular Atrophy:

Gait and Bone Health in SMA

A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Took Part in an Earlier Nusinersen Trial (ONWARD)

Progressive Rehabilitation Therapy in Patients With Advanced Lung Disease

COMMAND Early Feasibility Study: Implantable BCI to Control a Digital Device for People With Paralysis

Real-World Use of Novel Treatments in Patients With Spinal Muscular Atrophy (SMA): A Multi-Site Retrospective Chart Review of Pediatric SMA Patients Outside of the United States

A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients

A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec

Inherited Retinal Degenerative Disease Registry

Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases

Study of ALS Reversals 4: LifeTime Exposures

Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)

A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy

North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)

A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy

Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies

A Study to Assess FLX-787 in Subjects With Motor Neuron Disease Experiencing Muscle Cramps.

Effect of Functional Exercise in Patients With Spinal Bulbar Muscular Atrophy

A Study to Learn About the Investigational Drug Rinzimetostat (ORIC-944) in Patients With mCRPC Who Were Previously Treated With Abiraterone Acetate (Himalayas-1)