Welcome to Paid Research Studies




  • Conditions:   GM2 Gangliosidosis;   Tay-Sachs Disease;   Sandhoff Disease
    Intervention:   Drug: IB1001
    Sponsor:   IntraBio Inc
    Not yet recruiting

  • Conditions:   GM1 Gangliosidoses;   GM2 Gangliosidoses;   Tay-Sachs Disease;   Sandhoff Disease
    Interventions:   Drug: miglustat;   Other: Ketogenic Diet
    Sponsors:   University of Minnesota - Clinical and Translational Science Institute;   Rare Diseases Clinical Research Network;   National Center for Advancing Translational Science (NCATS);   National Institute of Neurological Disorders and Stroke (NINDS);   National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK);   Lysosomal Disease Network
    Recruiting

  • Conditions:   Tay-Sachs Disease;   Sandhoff Disease;   Late Onset Tay-Sachs Disease;   GM1 Gangliosidosis;   GM2 Gangliosidosis
    Intervention:  
    Sponsors:   University of Minnesota - Clinical and Translational Science Institute;   Rare Diseases Clinical Research Network;   National Center for Advancing Translational Science (NCATS);   National Institute of Neurological Disorders and Stroke (NINDS);   National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK);   Lysosomal Disease Network
    Recruiting

  • Conditions:   GM2 Gangliosidosis;   Supportive Care
    Intervention:   Drug: Miglustat
    Sponsors:   Tehran University of Medical Sciences;   Mashhad University of Medical Sciences;   Kashan University of Medical Sciences
    Recruiting

  • Conditions:   MLD;   Krabbe Disease;   ALD;   MPS I;   MPS II;   MPS III;   Vanishing White Matter Disease;   GM3 Gangliosidosis;   PKAN;   Tay-Sachs Disease;   NP Deficiency;   Osteopetrosis;   Alpha-Mannosidosis;   Sandhoff Disease;   Niemann-Pick Diseases;   MPS IV;   Gaucher Disease;   GAN;   GM1 Gangliosidoses;   Morquio Disease;   S-Adenosylhomocysteine Hydrolase Deficiency;   Batten Disease;   Pelizaeus-Merzbacher Disease;   Leukodystrophy;   Lysosomal Storage Diseases;   Purine Nucleoside Phosphorylase Deficiency;   Multiple Sulfatase Deficiency Disease
    Interventions:   Other: Palliative Care;   Biological: Hematopoetic Stem Cell Transplantation
    Sponsor:   University of Pittsburgh
    Recruiting

  • Conditions:   Adrenoleukodystrophy;   Batten Disease;   Mucopolysaccharidosis II;   Leukodystrophy, Globoid Cell;   Leukodystrophy, Metachromatic;   Neimann Pick Disease;   Pelizaeus-Merzbacher Disease;   Sandhoff Disease;   Tay-Sachs Disease;   Brain Diseases, Metabolic, Inborn;   Alpha-Mannosidosis;   Sanfilippo Mucopolysaccharidoses
    Intervention:   Biological: DUOC-01
    Sponsors:   Joanne Kurtzberg, MD;   The Marcus Foundation
    Recruiting

  • Conditions:   Neurological Regression;   Myoclonus;   Cherry Red Spot;   Brain Atrophy
    Intervention:  
    Sponsor:   National Human Genome Research Institute (NHGRI)
    Recruiting