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Batten Disease Clinical Trials

A listing of Batten Disease medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.


Found (15) clinical trials

Study of Ranolazine in Myotonia Congenita Paramyotonia Congenita and Myotonic Dystrophy Type 1

Recent advances in the understanding of myotonia congenita have identified potential areas that could possibly respond to treatment in a drug study. The drug ranolazine (trade name Ranexa) is a FDA-approved medication to treat chest pain in patients with heart disease. Ranolazine has been studied in mice with myotonia congenita. ...


Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry

The National Registry of DM and FSHD Patients and Family Members was developed to create a link between patients and scientists in order to promote research on these rare illnesses. The Registry is sponsored by the National Institutes of Health. Patients that are interested in joining the Registry can email ...

Phase N/A

Longitudinal Study of Neurodegenerative Disorders

Patients would be evaluated by a multidisciplinary team at specific time points every 3 months the first year, every 6 months the second year and once a year thereafter.

Phase N/A

Genetic Characterization of Movement Disorders and Dementias

Objective The objective of this study is to ascertain individuals with a clinical diagnosis of a movement disorder or dementia, their affected and unaffected family members, and unrelated, healthy individuals (to provide control samples); to characterize their phenotypes; and to identify and further characterize genetic contributions to etiology by collecting ...

Phase N/A

RAMYD Study - Evaluation of Arrhythmic Risk in Myotonic Dystrophy

Myotonic dystrophy type 1 (DM1, Steinert disease) is a multisystem disorder that affects, beside muscle, several other organs, including the heart. Cardiac involvement represents a major problem in the clinical management of patients, so that cardiac complications represent one of the primary causes of premature death in DM1. In particular ...


Inherited Retinal Degenerative Disease Registry

My Retina Tracker provides three different portals for data entry and review. An entry in My Retina Tracker is initiated by a participant, not a clinician. Using the Participant Portal, the participant establishes a username and password, is guided through on-line informed consent, and can then use an interactive guide ...

Phase N/A

A clinical trial seeking patients for a research study for the treatment of Tay-Sachs Disease, Metachromatic leukodystrophy, Hunter's Syndrome, Niemann-Pick Disease, Sandhoff Disease, Neuronal ceroid lipofuscinosis, Adrenoleukodystrophy, Krabbe's Disease, alpha-Mannosidosis, Sanfilippo Mucopolysaccharidoses, Brain Diseases, Metabolic, Inborn, Pelizaeus-Merzbacher Disease

The inherited metabolic disorders (IMD) are a heterogeneous group of genetic diseases, most of which involve a single gene mutation resulting in an enzyme defect. In the majority of cases, the enzyme defect leads to the accumulation of substrates that are toxic and/or interfere with normal cellular function. Often times, ...


Sleep Breathing Disorders a Main Trigger for Cardiac ARythmias in Type I Myotonic Dystrophy ?

Obstructive sleep apnea (OSA) and central sleep apnea (CSA), the most common form of sleep disordered breathing (SDB), are prevalent in patients with myotonic dystrophy type 1 (DM1). Among the serious complications from sleep apnea, the most alarming are cardiovascular, including arrhythmias and sudden cardiac death (SCD). Diagnosis of SDB ...

Phase N/A

Myotonic Dystrophy Family Registry

The Myotonic Dystrophy Family Registry (MDFR) is an online, patient-entered database that collects information on myotonic dystrophy (DM) such as disease symptoms and demographic information to aid researchers in developing new, effective treatments and help identify participants for research studies and clinical trials. The Registry supports trials and studies, making ...

Phase N/A

Clinical and Neuropsychological Investigations in Batten Disease

Batten Disease is an inherited disorder that causes progressive cognitive and behavioral decline in children. There have been no systematic clinical studies of Batten Disease using standardized rating instruments with known inter-rater reliability and validity. The Batten Study Group developed the Unified Batten Disease Rating Scale (UBDRS), a clinical rating ...

Phase N/A