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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.

RESULTS

Found (5) 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. ...

Phase

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 ...

Phase

Batten CLN6 Gene Therapy

The proposed clinical trial is the first human, open-label, single dose study of self-complementary AAV9 carrying the CLN6 gene under the control of a hybrid CMV enhancer/chicken--actin promoter (scAAV9.CB.CLN6) delivered one-time through an intrathecal catheter inserted by a lumbar puncture into the interspinous into the subarachnoid space of the lumbar ...

Phase

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

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, ...

Phase

Ventilatory Response After Non Invasive Ventilation in Type 1 Myotonic Dystrophy

Type 1 Myotonic Dystrophy is a hereditary neuromuscular disease with an autosomal dominant pattern whose prevalence is 1/8000 inhabitants and is the most common muscular dystrophy in adults. It is multisystem disease and is characterized by myotonia, progressive muscle loss and a wide spectrum of manifestations. Myotonic dystrophy type 1 ...

Phase N/A