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Queen Square Centre for Neuromuscular Diseases

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Jain Foundation natural history and clinical outcomes study of dysferlinopathy (limb-girdle muscular dystrophy type 2B)

Sponsor The Newcastle upon Tyne Hospitals NHS Foundation Trust
Funder Jain Foundation
CI Professor Kate Bushby
Sites Newcastle
More information

Introduction

This is a multi-centre assessment of 150 patients from centres for muscular dystrophy diagnosis and management. We will study a selection of possible outcome measures for dysferlinopathy trials.

Primary objective

To define the natural history of dysferlinopathy over three years.

Recruitment

Large unselected patient group with respect to age and nature of disease onset, progression and complications.

It will use existing and expanded registries and databases. The study will extend the existing registry activities co-ordinated by the Jain Foundation.