Rapamycin and Chloroquine: The In Vitro and In Vivo Effects of Autophagy-Modifying Drugs Show Promising Results in Valosin Containing Protein Multisystem Proteinopathy

Rapamycin: One Drug, Many Effects

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Rapamycin nanoparticles target defective autophagy in muscular dystrophy to enhance both strength and cardiac function

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Genetic Reduction of Mammalian Target of Rapamycin Ameliorates Alzheimer's Disease-Like Cognitive and Pathological Deficits by Restoring Hippocampal Gene Expression Signature

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Efficacy and Safety of Sirolimus for Renal Angiomyolipoma in Patients with Tuberous Sclerosis Complex or Sporadic Lymphangioleiomyomatosis: A Systematic Review

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Successful Treatment of Subungual Fibromas of Tuberous Sclerosis With Topical Rapamycin

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Exercise ameliorates the detrimental effect of chloroquine on skeletal muscles in mice via restoring autophagy flux

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Rapamycin-induced autophagy aggravates pathology and weakness in a mouse model of VCP-associated myopathy

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Inclusion body myopathy with paget disease of bone and frontotemporal dementia associated with a novel G156s mutation in theVCPgene

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mTOR Inhibition Alleviates Mitochondrial Disease in a Mouse Model of Leigh Syndrome

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mTOR dysfunction contributes to vacuolar pathology and weakness in valosin-containing protein associated inclusion body myopathy

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Autophagy: A double-edged sword in Alzheimer’s disease

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A progressive translational mouse model of human valosin-containing protein disease: TheVCPR155H/+mouse

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The Homozygote VCPR155H/R155H Mouse Model Exhibits Accelerated Human VCP-Associated Disease Pathology

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Slow development of ALS-like spinal cord pathology in mutant valosin-containing protein gene knock-in mice

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Autophagy as a new therapeutic target in Duchenne muscular dystrophy

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Autophagy in Lysosomal Myopathies

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Fighting neurodegeneration with rapamycin: mechanistic insights

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Autophagy gone awry in neurodegenerative diseases

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Two Australian families with inclusion-body myopathy, Paget’s disease of bone and frontotemporal dementia: Novel clinical and genetic findings

(2010) Kishore R. Kumar et al.   NEUROMUSCULAR DISORDERS

Exome Sequencing Reveals VCP Mutations as a Cause of Familial ALS

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VCP Associated Inclusion Body Myopathy and Paget Disease of Bone Knock-In Mouse Model Exhibits Tissue Pathology Typical of Human Disease

(2010) Mallikarjun Badadani et al.   PLoS One

Valosin-containing protein (VCP) is required for autophagy and is disrupted in VCP disease

(2009) Jeong-Sun Ju et al.   JOURNAL OF CELL BIOLOGY

Inclusion body myopathy with Paget disease and frontotemporal dementia (IBMPFD): clinical features including sphincter disturbance in a large pedigree

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A novel mutation in the VCP gene (G157R) in a german family with inclusion-body myopathy with paget disease of bone and frontotemporal dementia

(2009) Atbin Djamshidian et al.   MUSCLE & NERVE

Valosin containing protein associated inclusion body myopathy: abnormal vacuolization, autophagy and cell fusion in myoblasts

(2009) Jouni Vesa et al.   NEUROMUSCULAR DISORDERS

Clinical studies in familial VCP myopathy associated with Paget disease of bone and frontotemporal dementia

(2008) Virginia. E. Kimonis et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Autophagy in the Pathogenesis of Disease

(2008) Beth Levine et al.   CELL

Impaired Protein Aggregate Handling and Clearance Underlie the Pathogenesis of p97/VCP-associated Disease

(2008) Jeong-Sun Ju et al.   JOURNAL OF BIOLOGICAL CHEMISTRY