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Returning 66 results

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

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