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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 (6) clinical trials

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

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

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

Impact of P53 and SIRT1 in Type 2 Diabetes

Investigating the impact of p53 and SIRT1 in the development of type 2 DM through the treatment of prediabetic individuals by either nigetella salivata or Metformin

Phase

Clinical Study Evaluating Vildagliptin Versus Vildagliptin/Metformin on NAFLD With DM

Clinical study evaluating vildagliptin versus vildagliptin/metformin on nonalcoholic fatty liver disease with DM.

Phase

Safety Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1

This study is evaluating single administration of two dose levels of ERX-963 to explore the relationship between dose, safety, tolerability, exposure and clinical benefit. This is a multi-center, randomized, double-blind, placebo-controlled, two-treatment period crossover study in two cohorts of participants with DM1. Participants who have consented and meet eligibility criteria ...

Phase