Genetic Disease

Long-term Cardiac Monitoring in Epilepsy

Conditions:   Focal Epilepsy;   Intractable Epilepsy;   Epileptic Encephalopathy;   Lennox Gastaut Syndrome;   Symptomatic Epilepsy;   Generalized Epilepsy
Intervention:   Device: Reveal LINQ ICM System
Sponsors:   Northwell Health;   Medtronic
Recruiting
ClinicalTrials.gov: Genetic_Disease | Recruiting, Not yet recruiting, Available Studies | United States | First posted in the last 100 days

May 20, 2019 / by / in
Sickle Cell Disease, Hemechip

Condition:   Sickle Cell Disease
Intervention:   Device: HemeChip
Sponsors:   University Hospitals Cleveland Medical Center;   US federal government;   Amino Kano Teaching Hospital;   Murtala Mohammed Specialist Hospital;   Hasiya Bayero Pediatric Hospital;   University of Nebraska;   University of North Carolina, Chapel Hill;   Case Western Reserve University
Recruiting
ClinicalTrials.gov: Genetic_Disease | Recruiting, Not yet recruiting, Available Studies | United States | First posted in the last 100 days

May 19, 2019 / by / in
A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type II GM1 Gangliosidosis

Conditions:   Lysosomal Diseases;   Gangliosidosis;   GM1
Intervention:   Biological: AAV9-GLB1
Sponsor:   National Human Genome Research Institute (NHGRI)
Recruiting
ClinicalTrials.gov: Genetic_Disease | Recruiting, Not yet recruiting, Available Studies | United States | First posted in the last 100 days

May 16, 2019 / by / in
Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)

Conditions:   Facioscapulohumeral Muscular Dystrophy;   Charcot-Marie-Tooth Disease
Intervention:   Drug: ACE-083
Sponsor:   Acceleron Pharma, Inc.
Recruiting
ClinicalTrials.gov: Genetic_Disease | Recruiting, Not yet recruiting, Available Studies | United States | First posted in the last 100 days

May 12, 2019 / by / in