P281: JOURNEY: A natural history study of limb-girdle muscular dystrophies R3–R5, baseline characteristics of study cohort

• Linda P. Lowes,
• Carlos Ortez Gonzalez,
• Kristl Claeys,
• Chamindra Laverty,
• Andrea Gangfuss,
• Crystal Proud,
• Jordi Diaz Manera,
• Meredith James,
• Lindsay Alfano,
• Herb Stevenson,
• Lixi Yu,
• André Mueller-York,
• Giacomo Comi

Affiliations

Linda P. Lowes

Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital, Columbus, OH

Carlos Ortez Gonzalez

Neuromuscular Unit, Hospital Sant Joan de Deu, Barcelona, Spain

Kristl Claeys

Department of Neurology, University Hospitals Leuven and KU Leuven, Leuven, Belgium

Chamindra Laverty

University of California San Diego, San Diego, CA

Andrea Gangfuss

Department of Paediatric Neurology, Centre for Neuromuscular Disorders, Centre for Translational Neuro- and Behavioral Sciences, University of Duisberg-Essen, Essen, Germany

Crystal Proud

Children’s Hospital of The King’s Daughter, Norfolk, VA

Jordi Diaz Manera

Children’s Hospital of The King’s Daughter, Norfolk, VA

Meredith James

The John Walton Muscular Dystrophy Research Center, Newcastle University, Newcastle Upon Tyne Hospital

Lindsay Alfano

Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital, Columbus, OH

Herb Stevenson

Sarepta Therapeutics, Inc., Cambridge, MA

Lixi Yu

Sarepta Therapeutics, Inc., Cambridge, MA

André Mueller-York

Sarepta Therapeutics, Inc., Cambridge, MA

Giacomo Comi

Dino Ferrari Center, Department of Pathophysiology and Transplantation, University of Milan