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Long-Term Development of Muscular Dystrophy Outcome Assessments

Description

This is a 24-month, observational study of up to 1000 participants with Limb Girdle Muscular Dystrophy (LGMD), Myotonic Dystrophy Type 2 (DM2), and late onset Pompe disease (LOPD).

Conditions

LGMD1BLGMD1CLGMD1DLGMD1ELGMD1FLGMD1GLGMD1HLGMD2ALGMD2BLGMD2CLGMD2DLGMD2ELGMD2FLGMD2GLGMD2ILGMD2JLGMD2KLGMD2LLGMD2MLGMD2NLGMD2OLGMD2PLGMD2QLGMD2SLGMD2TLGMD2ULGMD2WLGMD2XLGMD2Y
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Related Conditions:

LGMD1BLGMD1CLGMD1DLGMD1ELGMD1FLGMD1GLGMD1HLGMD2ALGMD2BLGMD2CLGMD2DLGMD2ELGMD2FLGMD2GLGMD2ILGMD2JLGMD2KLGMD2LLGMD2MLGMD2NLGMD2OLGMD2PLGMD2QLGMD2SLGMD2TLGMD2ULGMD2WLGMD2XLGMD2Y

Study Overview

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Study Details

Study overview

This is a 24-month, observational study of up to 1000 participants with Limb Girdle Muscular Dystrophy (LGMD), Myotonic Dystrophy Type 2 (DM2), and late onset Pompe disease (LOPD).

Long-Term Development of Muscular Dystrophy Outcome Assessments (GRASP-01-005)

Long-Term Development of Muscular Dystrophy Outcome Assessments

Condition
LGMD1B
Intervention / Treatment

-

Contacts and Locations

Richmond

Virginia Commonwealth University, Richmond, Virginia, United States, 23298

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. Age between 6-50 years at enrollment
  • 2. Clinically affected (defined as weakness on bedside evaluation in a pattern consistent with proximal weakness)
  • 3. Genetic confirmation of a LGMD, DM2, or LOPD
  • 4. FVC above 30% of predicted
  • 1. Any other illness that would interfere with the ability to undergo safe testing or would interfere with interpretation of the results in the opinion of the site investigator
  • 2. Participation in a clinical trial receiving an investigational product

Ages Eligible for Study

6 Years to 50 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Virginia Commonwealth University,

Nicholas Johnson, MD, PRINCIPAL_INVESTIGATOR, Virginia Commonwealth University

Study Record Dates

2029-05-01