Work

Outcomes of Autologous Hematopoietic Stem Cell Transplant in Sporadic Late Onset Nemaline Myopathy with Associated Monoclonal Gammopathy of Unknown Significance

Year: 2018

Type: article

Abstract: Background: Sporadic Late Onset Nemaline Myopathy (SLONM) is a rare muscle disorder with subacute progression, characterized by axial and proximal muscle weakness and atrophy, and the presence of nemaline rods in myofibers. When SLONM is associated with a monoclonal gammopathy of undetermined significance (SLONM/MGUS), patients seem to fare worse compared to patients with SLONM and no MGUS. Supportive and immunosuppressive therapies have yielded poor outcomes for most patients with SLONM/MGUS; IVIG have been beneficial in some.

Source: Transplantation and Cellular Therapy

DOI: 10.1016/j.bbmt.2017.12.065