Abstract: Compounds represented by general Formulae I to IV as described herein are disclosed. Further disclosed are composition utilizing the herein disclosed compounds and using the same for the treatment of glycogen storage disorders.

Abstract: Compounds represented by general Formulae I to IV as described herein are disclosed. Further disclosed are composition utilizing the herein disclosed compounds and using the same for the treatment of glycogen storage disorders.

Abstract: Disclosed herein is a polypeptide including a pharmaceutical composition for use in prevention or treatment of a disease or a disorder associated with lysosomal storage and an autophagy-misregulation associated disease. Further provided are agents that bind a region of an N-terminal domain of a lysosomal-associated membrane protein 1 (LAMP-1), and methods for treating or preventing development of a disease or a disorder associated with lysosomal storage, polyglucosan accumulation or abnormal glycogen accumulation and autophagy-misregulation in a subject in need thereof.

Abstract: Compounds represented by general Formulae I to IV as described herein are disclosed. Further disclosed are composition utilizing the herein disclosed compounds and using the same for the treatment of glycogen storage disorders.

Abstract: Compounds represented by general Formulae I to IV as described herein are disclosed. Further disclosed are composition utilizing the herein disclosed compounds and using the same for the treatment of glycogen storage disorders.

Abstract: Compounds represented by general Formulae I to IV as described herein are disclosed. Further disclosed are composition utilizing the herein disclosed compounds and using the same for the treatment of glycogen storage disorders.

Abstract: Compounds represented by general Formulae I to IV as described herein are disclosed. Further disclosed are composition utilizing the herein disclosed compounds and using the same for the treatment of glycogen storage disorders.

Abstract: The current invention is a method of preventing and treating certain diseases by preventing the synthesis and/or breakdown of glycogen by the administration of an agent, guaiacol. Diseases that can be prevented and treated by the administration of guaiacol include but are not limited to glycogen storage disease type IV (GSD-IV), adult polyglucosan body disease (APBD), and Lafora disease (LD). The invention also includes methods and tools for screening for agents that prevent and treat these diseases as well as basic research, specifically in the form of cells and cell lines that produce detectable polyglucosan.

Abstract: Compounds represented by general Formulae I to IV as described herein are disclosed. Further disclosed are composition utilizing the herein disclosed compounds and using the same for the treatment of glycogen storage disorders.

Abstract: A method of diagnosing amyotrophic lateral sclerosis (ALS) in a subject in need thereof is provided. The method comprising determining in cells of the subject at least one alteration in a sequence or expression level of Cytoplasmic FMR Interacting Protein (CyFIP2; Accession AF160973) and/or Retinoblastoma Binding Protein 9 (RbBP9; Accession AF039564), wherein an altered sequence or expression of the sequence or expression level of the CyFIP2; Accession AF160973) or Retinoblastoma Binding Protein 9 (RbBP9; Accession AF039564) as compared to a control reference sample from a non-ALS subject, is indicative of ALS. The present teachings may be implemented in screening for novel anti-ALS medicaments and for treating ALS.