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Muscular Dystrophy Clinical Trials

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

Found 136 clinical trials
K Kayleigh Worek, Program Manager, MS

BetterLife FSHD: A Patient-driven Health and Research Platform

BetterLife FSHD is a registry platform built to support people living with FSHD. It connects patients with personalized resources, tools, and research opportunities that match their health experiences and needs. At the same time, BetterLife collects secure health and experience data from patients to support research. This data is shared …

1 years of age All Phase N/A
A Aurore Daron

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45

This is a study of the investigational medicine ENTR-601-45 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition. The researchers want to: Test how safe ENTR-601-45 is, learn about any side effects, and look at the potential positive effects of ENTR-601-45, compared to placebo. Placebo looks like …

4 - 20 years of age Male Phase 1/2
C Casie Genetti, MS, LCGC

Molecular Analysis of Patients With Neuromuscular Disease

The purpose of this study is to identify new genes responsible for neuromuscular disorders and study muscle tissue of patient with known neuromuscular disease, as well as their family members. We are interested in recruiting many types of neuromuscular disease including; Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD), and …

1 - 100 years of age All Phase N/A
S Stephanie Thera

Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)

The primary goal of this proposal is to collect motor and functional outcomes specific to FSHD over time. By collecting measures specific to FSHD, this will help ensure the best level of clinical care is being provided. Also, the hope is to speed up drug development by gaining a better …

years of age All Phase N/A
R Rachel Alvarez, BS

Congenital Muscle Disease Study of Patient and Family Reported Medical Information

The Congenital Muscle Disease Patient and Proxy Reported Outcome Study (CMDPROS) is a longitudinal 10 year study to identify and trend care parameters, adverse events in the congenital muscle diseases using the Congenital Muscle Disease International Registry (CMDIR) to acquire necessary data for adverse event calculations (intake survey and medical …

years of age All Phase N/A
L Lorna McNeill

Understanding Patient Experience Among Asians at MD Anderson

The purpose of this research study is to understand barriers to and facilitators of a positive patient experience for Asian patients.

18 years of age All Phase N/A
C Claudia E Kuehni, Prof. MD

Swiss Registry for Neuromuscular Disorders

The Swiss Patient Registry for DMD/BMD and SMA was launched in 2008 in order to give Swiss patients access to new therapies. It was founded with the financial support of several patient organizations and research foundations. Since 2008, children, adolescents and adults with DMD, BMD and SMA are registered with …

years of age All Phase N/A

Molecular Testing for the MD Anderson Cancer Center Personalized Cancer Therapy Program

This study performs standardized testing of tumor tissue samples to learn which genes are mutated (have changed) in order to provide personalized cancer therapy options to cancer patients at MD Anderson. This may help doctors use testing information on tumors to identify clinical trials that may be most relevant to …

years of age All Phase N/A
S Samoel KHAMADI, Dr

Clinical Performance Evaluation of MagIA IVD-MD Multiplex Testing (HIV/HBV/HCV/Syphilis)

Performance study to evaluate the clinical performance of the In-Vitro Diagnostics Medical Device MagIA H3S (a Multiplex Point-of-Care test for the combined detection of Human Immunodeficiency Virus (HIV), Hepatitis B and C and Syphilis) from serum, plasma samples collected prospectively or retrospectively in Ivory Coast and Kenya.

18 years of age All Phase N/A
R Registry Project Manager and Curator

The United Kingdom National Registry for Myotonic Dystrophy

Myotonic dystrophy (dystrophia myotonica - DM) exists in two forms, usually referred to as DM1 (type 1) and DM2 (type 2). Both conditions are genetic disorders but each affects a different gene. DM1 is the most common adult-onset muscular dystrophy, and is thought to affect at least 1 in 8,000 …

years of age All Phase N/A

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