🔬 Current Disease Focus:
Task Progress: 0%
👥 Active Problem Solvers
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🌍 Experts collaborating globally
⚡ Current Task Assignment
🧬 Genetic Analysis
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📊 Resource Allocation by Research Phase:
📚 Data Analysis
🧪 Experimental Design
🔬 Clinical Trials
💊 Treatment Development
This is a long-term follow-up safety and efficacy study of participants in ...
Status
Active Not Recruiting
Location
Stanford University Medical Center
Start Date
2020-02-10
Trial ID
NCT04042025
Treatments Being Tested
The purpose of this trial is to evaluate the safety and tolerability of int...
Status
Terminated
Location
UCLA
Start Date
2017-12-14
Trial ID
NCT03381729
Treatments Being Tested
With the advent of new treatments for ASI, new phenotypes are emerging. The...
Status
Recruiting
Location
Pediatric Rehabilitation Service - L'Escale Mother and Child Hospital
Start Date
2024-07-24
Trial ID
NCT06321965
In this study, researchers will learn more about the use of nusinersen (BII...
Status
Completed
Location
Arkansas Children's Hospital Research Institute
Start Date
2021-01-04
Trial ID
NCT04488133
Treatments Being Tested
To evaluate the safety and efficacy of intravenous onasemnogene abeparvovec...
Status
Completed
Location
David Geffen School of Medicine at UCLA
Start Date
2018-04-02
Trial ID
NCT03505099
Treatments Being Tested
This study aims to assess the safety and effectiveness of onasemnogene abep...
Status
Not Yet Recruiting
Location
Multiple Locations
Start Date
2026-10-30
Trial ID
NCT07767890
This is a Phase 3, open-label, single-arm, single-dose, trial of onasemnoge...
Status
Completed
Location
Tokyo Women's Medical University
Start Date
2019-05-31
Trial ID
NCT03837184
Treatments Being Tested
The natural history of SMA patients has changed, due to the improvements in...
Status
Unknown
Location
Hospital Italiano de Buenos Aires
Start Date
2022-08-17
Trial ID
NCT05475691
A long-term safety study in Brazilian patients with a confirmed diagnosis o...
Status
Recruiting
Location
Novartis Investigative Site
Start Date
2023-11-22
Trial ID
NCT06019637
Treatments Being Tested
In this study, researchers will learn more about the safety and effects of ...
Status
Recruiting
Location
Neurology Rare Disease Center
Start Date
2026-09-04
Trial ID
NCT07444450
Treatments Being Tested