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Study Title![]() |
Location | Status | Disorders |
---|---|---|---|
A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I | International Locations | Completed | Cognitive Decline, Mucopolysaccharidosis I, Hurler-Scheie Syndrome, Scheie Syndrome, Lysosomal Storage Disease |
Advancing Research and Treatment for Frontotemporal Lobar Degeneration (ARTFL) | Multiple U.S. Locations | Recruiting | FTLD, Progressive Supranuclear Palsy (PSP), Frontotemporal Dementia (FTD), Corticobasal Degeneration (CBD), PPA Syndrome, Behavioral Variant Frontotemporal Dementia (bvFTD), Semantic Variant Primary Progressive Aphasia (svPPA), Nonfluent Variant Primary Progressive Aphasia (nfvPPA), FTD With Amyotrophic Lateral Sclerosis (FTD/ALS), Amyotrophic Lateral Sclerosis (ALS), Oligosymptomatic PSP (oPSP), Corticobasal Syndrome (CBS) |
AFQ056 for Language Learning in Children With FXS | No documented Location | Not Yet Recruiting | Fragile X Syndrome |
Alpers Huttenlocher Natural History Study | Multiple U.S. Locations | Recruiting | Alpers Huttenlocher Syndrome |
An Analysis of the Symptomatic Domains Most Relevant to Charcot Marie Tooth Neuropathy (CMT) Patients | Florida | Completed | Charcot Marie Tooth Disease (CMT), Hereditary Sensory and Motor Neuropathy, Nerve Compression Syndromes, Tooth Diseases, Congenital Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System |
Androgen Effect on Klinefelter Syndrome Motor Outcome | Pennsylvania | Completed | Klinefelter Syndrome |
Assessment of N-Acetylcysteine as Therapy for Myalgic Encephalomyelitis/Chronic Fatigue Syndrome | New York | Not Yet Recruiting | Chronic Fatigue Syndrome, Myalgic Encephalomyelitis |
Biobanking of Rett Syndrome and Related Disorders | Multiple U.S. Locations | Recruiting | Rett Syndrome, MECP2 Duplication, CDKL5, FOXG1 Disorders |
Biomarker Development in Sturge-Weber Syndrome | Maryland | Completed | Sturge-Weber Syndrome |
Botulinum Toxin to Treat Restless Legs Syndrome | Maryland* | Completed | Restless Legs Syndrome |