Description: This special interest group session will focus on two areas of contemporary interest in neuromuscular disease. One session will review familial amyloid polyneuropathy and examine the recent development and release of several novel therapies that improve the natural history of this disorder. Another session will review the expanding number of recognized autoantibodies in myositis and discuss the implications for diagnosis and treatment in this heterogeneous group of autoimmune diseases. Three short presentations have been selected from the best accepted neuromuscular abstracts.

Chair: Vern Juel, MD, Duke University

Co-Chair: Michelle Mauermann, MD, Mayo Clinic

Objective(s):

• Recognize the clinical features of familial amyloid polyneuropathy
• Give examples of contemporary treatments for familial amyloid polyneuropathy
• Recognize the limitations of treatments for familial amyloid polyneuropathy
• Identify common myositis autoantibodies
• Describe treatment strategies for distinct autoimmune myopathy syndromes

Presentations:

Therapeutic Advances in Hereditary Transthyretin Amyloid Polyneuropathy

Speaker:

Michelle Mauermann, MD

Mayo Clinic

Myositis Immunology and Autoantibodies

Speaker:

Andrew Mammen, MD, PhD

National Institutes of Health

Neurotoxic Properties of Human Endogenous Retrovirus-k Envelope Protein and Detection in CSF of Patients with ALS

Oral Presenter:

Avindra Nath, MD

National Institutes of Health

Geographical Variation in Proportion of MuSK Antibody Myasthenia Gravis Around the World – A Multicenter Study

Oral Presenter:

Angela Vincent, MBBS

University of Oxford

NADPH Oxidase 5: A New Player in Peripheral Neuropathy

Oral Presenter:

Stephanie Eid, PhD

University of Michigan