Arimoclomol, the company's lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, sporadic Inclusion Body Myositis
, and Amyotrophic Lateral Sclerosis.
Kimura, "Inclusion body myositis
: expression of extracellular signal-regulated kinase and its substrate," Neurology, vol.
* The report reviews key players involved Inclusion Body Myositis
(IBM) therapeutics and enlists all their major and minor projects
* The report reviews key players involved in the therapeutics development for Inclusion Body Myositis
(IBM) and enlists all their major and minor projects
(6) Conversely, CK is often normal or only mildly elevated in inclusion body myositis
. Up to 80% of patients with autoimmune myopathy will have antinuclear antibodies.
Sporadic inclusion body myositis
: variability in prevalence and phenotype and influence of the MHC.
Hilton-Jones, "Clinical assessment determines the diagnosis of inclusion body myositis
independently of pathological features," Journal of Neurology, Neurosurgery & Psychiatry, vol.
BOSTON -- Failure to respond to standard immunosuppressant therapy may be the first sign that a patient's apparent polymyositis actually is inclusion body myositis
, according to Dr.
Inclusion Body Myositis
causes a gradual deterioration of muscle strength and can affect most limb muscles.
Diseases affecting muscles include polymyositis, dermatomyositis and inclusion body myositis
. The main symptom is muscle weakness in the upper arms, upper legs and neck, muscle pain, fatigue, joint pain and swelling, rashes over the face and knuckles, fevers, swallowing difficulty and shortness of breath.
They are contrasted with other types of inflammatory myopathies, including inclusion body myositis
and myositis caused by infection or drug reactions.
Orphazyme is testing arimoclomol in four indications including amyotrophic lateral sclerosis, Niemann-Pick disease Type C, Gaucher disease and sporadic Inclusion Body Myositis