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I-8 Muscle maturation and early pathogenic findings in spinal muscular atrophy: any clues for therapy?

Pacini Editore SpA
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  • Lectures By Invited Speakers
  • Biology
  • Medicine


Muscle maturation and early pathogenic findings in spinal muscular atrophy: any clues for therapy? 154 10th Congress of The Mediterranean Society of Myology, April 28-30, 2011 tain antibodies play a role by recruiting macrophages to the muscle in an ADCC process, remains unclear. In uncontrolled studies, PM and DM respond to prednisone to some degree and for some period of time; adding an immuno- suppressive drug (Azathioprine, Cyclosporine, Mycophenolate, Methotrexate) may have a steroid-sparing effect but their benefit is uncertain. In contrast, IBM is resistant to most of these thera- pies, most times. Controlled studies have shown that IVIg is effective and safe for the treatment of DM. The clinical benefit, which can be impressive in patients with early disease, is associ- ated with improvement in the muscle cytoarchitecture and reso- lution of the aberrant immunopathological parameters, includ- ing interception of complement activation and downregulation of ICAM-I, VCAM, TGF-β, MHC-I and various immunoregu- latory and structural genes. IVIg seems to be also effective in patients with PM but offers transient help to a small number of patients with IBM. New agents currently on the market may be promising new therapies for the treatment of inflammatory myopathies. Among them include the monoclonal antibodies or fusion proteins against: a) molecules associated with T-cell-signaling path- ways, such as the anti-CD52 (CAMPATH), anti-LFA/ICAM (Leukocyte Functional Antigen/ Intracellular Adhesion Mol- ecule), and anti-IL2 receptor (IL2-receptor antagonist). Further, two cytophillin-binding drugs, Tacrolimus and Rapamycin, that prevent the IL2-induced T cell proliferation or transcription, may be candidate agents for certain conditions; b) B cells using the monoclonal antibody directed against CD20, expressed on B cells (Rituximab) or the humanized version Occrelizumab; agents against B-cell growth factors, such as BAFF and APRIL are in the offing; c) Complement C5

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