Sporadic late-onset nemaline myopathy effectively treated by melphalan and stem cell transplant.
Voermans
N C
NC
Neuromuscular Centre Nijmegen, Nijmegen Medical Centre, Radboud University, the Netherlands.
Minnema
M
M
Lammens
M
M
Schelhaas
H J
HJ
Kooi
A V D
AV
Lokhorst
H M
HM
van Engelen
B G
BG
D000328:Adult; D003937:Diagnosis, Differential; D006801:Humans; D008297:Male; D008558:Melphalan / Q000627:therapeutic use*; D016472:Motor Neuron Disease / Q000503:physiopathology; D019653:Myeloablative Agonists / Q000627:therapeutic use*; D017696:Myopathies, Nemaline / Q000150:complications / Q000503:physiopathology / Q000628:therapy*; D009220:Myositis / Q000503:physiopathology; D010265:Paraproteinemias / Q000150:complications; D033581:Stem Cell Transplantation
D019653:Myeloablative Agonists; D008558:Melphalan