Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD)

        Study Status: Open to enrollment

    Patient Population:

o   LGMD2A (CAPN3)

o   LGMD2B (DYSF)

o   LGMD2L (ANO5)

o   LGMD1D (DNAJB6)

o   LGMD2D (SGCA)

o   LGMD2E (SGCB)

o   LGMD2C (SGCD)

o   LGMD2F (SGCG)

·         Participation Duration: 12 months

·         Active Sites:

o   Virginia Commonwealth University

o   University of Kansas

o   Nationwide Children’s Hospital

o   University of California, Irvine

o   University of Colorado

o   University of Iowa

o   Washington University in St. Louis

o   Kennedy Krieger Institute

o   Newcastle University

o  University of Minnesota

·         Study Summary:

This is a non-interventional study observing the natural progression of LGMD in patients over the course of 12 months. The study’s primary goal is to identify standardized clinical outcome assessments, in the form of functional measures, to hasten therapeutic development.

 

Defining Endpoints in Becker Muscular Dystrophy

·         Study Status: Open to enrollment

·         Patient Population: Becker Muscular Dystrophy (BMD)

·         Participant Duration: 24 months

·         Active Sites:

o   Virginia Commonwealth University

o  University of Kansas

o  Nationwide Children’s Hospital

o  University of California, Irvine

o  University of Colorado

o  University of Iowa

o  Washington University in St. Louis

o  Kennedy Krieger Institute

o  University of Minnesota

·         Study Summary:

This is a non-interventional study observing the natural progression of BMD in patients over the course of 24 months. The study’s primary goal is to assess the natural history of muscle function in these patients as well as the impact of BMD on a participant’s quality of life.

 

Biomarker Development in LGMD2i

·         Study Status: closed to enrollment

·         Patient Population: LGMD2i (FKRP)

·         Participation Duration: 12 months

·         Active Sites:

o   Virginia Commonwealth University

o   University of Kansas

o   Nationwide Children’s Hospital

o   University of California, Irvine

o   University of Colorado

o   University of Iowa

o   Washington University in St. Louis

o   University of Florida

o   Kennedy Krieger Institute

o   Atrium Health

o   University of Copenhagen

Active Studies