Molecular Analysis of Patients With Neuromuscular Disease

Description

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 limb-girdle muscle dystrophy (LGMD). There are still many patients diagnosed with muscular dystrophy with no causative gene implicated in their disease. Using molecular genetics to unravel basis of these neuromuscular disorders will lead to more accurate diagnosis/prognosis of these disorders which will lead to potential therapies.

Conditions

Neuromuscular; Disorder, Hereditary, Duchenne/Becker Muscular Dystrophy, Limb-girdle Muscular Dystrophy

Study Overview

Study Details

Study overview

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 limb-girdle muscle dystrophy (LGMD). There are still many patients diagnosed with muscular dystrophy with no causative gene implicated in their disease. Using molecular genetics to unravel basis of these neuromuscular disorders will lead to more accurate diagnosis/prognosis of these disorders which will lead to potential therapies.

Molecular Analysis of Nucleic Acids Derived From Patients With Neuromuscular Disease and Their Family Members

Molecular Analysis of Patients With Neuromuscular Disease

Condition
Neuromuscular; Disorder, Hereditary
Intervention / Treatment

-

Contacts and Locations

Boston

Boston Children's Hospital, Boston, Massachusetts, United States, 02115

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • 1. having a clinical and/or pathological diagnosis of a muscular dystrophy
  • 2. being the first degree relative of someone with such a diagnosis
  • 3. having had a muscle biopsy if diagnosed with a neuromuscular disease
  • 4. willingness to provide a skin biopsy for research only
  • 1. not having a neuromuscular diagnosis in you or a family member
  • 2. not wishing to participate
  • 3. being incapable of giving consent and not having a legal guardian willing or able to do so

Ages Eligible for Study

1 Week to 100 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Boston Children's Hospital,

Louis M Kunkel, PhD, PRINCIPAL_INVESTIGATOR, Boston Children's Hospital/Harvard Medical School

Study Record Dates

2027-12-31