🔬 Current Disease Focus:
Task Progress: 0%
👥 Active Problem Solvers
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🌍 Experts collaborating globally
⚡ Current Task Assignment
🧬 Genetic Analysis
🔄 Matching experts to specific tasks
📊 Resource Allocation by Research Phase:
📚 Data Analysis
🧪 Experimental Design
🔬 Clinical Trials
💊 Treatment Development
Methylmalonic acidemia (MMA) is a rare genetic disorder caused by mutations...
Status
Terminated
Location
ZNA Queen Paola Child Hospital and Provincial Centre for Metabolic Disorders
Start Date
2010-07-19
Trial ID
NCT01141075
Treatments Being Tested
This study is being performed as a post-approval safety study (PASS), per t...
Status
Completed
Location
Krankenhaus der Barmherzigen Schwestern Linz
Start Date
2015-04-30
Trial ID
NCT02369731
In some participants with cystic fibrosis (CF), the disease is caused by a ...
Status
Completed
Location
Hadassah University Hospital - Mount Scopus
Start Date
2005-11-30
Trial ID
NCT00237380
Treatments Being Tested
This is an open-label extension study for participants who completed a Phas...
Status
Terminated
Location
University of Alabama at Birmingham
Start Date
2015-08-31
Trial ID
NCT02456103
Treatments Being Tested
This is a Phase 3, international, multicenter, randomized, double-blind, pl...
Status
Completed
Location
University of Alabama at Birmingham
Start Date
2014-08
Trial ID
NCT02139306
Treatments Being Tested
Single Center, open label, Phase I-II trial designed to test the safety and...
Status
Recruiting
Location
Amsterdam UMC, AMC
Start Date
2019-08-01
Trial ID
NCT04014530
Treatments Being Tested
This is a phase 2, crossover study of Ataluren for the treatment of nonsens...
Status
Completed
Location
New York University School of Medicine
Start Date
2016-11
Trial ID
NCT02758626
This study is designed to evaluate the ability of ataluren to increase dyst...
Status
Completed
Location
Phoenix Childrens Hospital
Start Date
2018-12-21
Trial ID
NCT03648827
Treatments Being Tested
Duchenne/Becker muscular dystrophy (DBMD) is a genetic disorder that develo...
Status
Completed
Location
Royal Children's Hospital
Start Date
2012-05-20
Trial ID
NCT01557400
Treatments Being Tested
Duchenne muscular dystrophy (DMD) is a genetic disorder that develops in bo...
Status
Terminated
Location
Cincinnati Children's Hospital Medical Center
Start Date
2008-08-13
Trial ID
NCT00759876
Treatments Being Tested