Involvement of Androgen/Androgen Receptor Pathway in Fabry Disease

A method of monitoring the efficacy of a therapeutic intervention on a patient suffering from Fabry's disease comprising the steps of:

• administering one or more pharmaceutical compositions to the patient at one or more pre-defined intervals, wherein the pharmaceutical composition comprises one or more therapeutic agents against Fabry's disease;
• collecting a sample from the patient;
• comparing a level of one or more molecules selected from the group consisting of an androgen, an androgen precursor, an androgen metabolite, an androgen-receptor (AR) pathway molecule, and one or more AR target genes and related metabolites in the sample from the patient undergoing the therapeutic intervention with that of the same patient prior to the commencement of the therapeutic intervention;
• and
• determining whether the therapeutic intervention is effective based on the comparison of a measured level of the one of more molecules in the sample from the patient before and after the commencement of the therapeutic intervention.

A pharmaceutical composition for treating Fabry's disease in a patient comprising:

• a therapeutically effective amount of at least one of an androgen synthesis blocker, a 5α-reductase enzyme inhibitors, an androgen-receptor (AR) gene expression knockdown agent, an inhibitor of HSP90, an AR pathway kinase blocking agents or a histone deacetylases inhibitors sufficient to treat Fabry's disease dissolved, dispersed or suspended in an aqueous or a non-aqueous solvent;
• one or more optional related co-factors, proteins, antibodies, pain medications, and other pharmaceutically active agents dissolved, dispersed, or suspended in an aqueous or a non-aqueous solvent;
• and
• one or more optional excipients, fillers, diluents, extended or controlled release agents, bulking agents, antiadherents, binders, lubricants, preservatives or any combinations thereof.