Why Diagnose and Treat Memory Loss, Alzheimer’s Disease, and Dementia?
A 72-year-old woman comes into the clinic at the urging of her son. She has noticed some difficulties finding words for the past six months, but denies problems with memory…
Dementia with Lewy Bodies (Including Parkinson’s Disease Dementia)
A 65-year-old man came to the clinic complaining of cognitive difficulties. He had been forgetful for about a year, often confused, and was unable to use the television remote. When…
Alzheimer’s Disease Dementia and Mild Cognitive Impairment Due to Alzheimer’s Disease
(See Chapters 2 and 3 for additional information.) Quick Start 1 Alzheimer’s Disease Definition • Alzheimer’s disease is a neurodegenerative disease of the brain characterized by a clinical dementia with…
Approach to the Patient with Memory Loss, Mild Cognitive Impairment, or Dementia
In this chapter we propose a strategy for evaluating patients with concerns about their memory and/or other aspects of cognition that will lead to a diagnosis and treatment plan. Quick…
Gene Therapy for Spinal Muscular Atrophy
Abstract Spinal muscular atrophy (SMA) is a devastating neurodegenerative disease that impacts approximately 1:10,000 live births. SMA is caused by deletion or loss-of-function mutations in the Survival Motor Neuron 1…
Stem Cell Therapy for Amyotrophic Lateral Sclerosis
Abstract Amyotrophic lateral sclerosis (ALS) is a challenging disease characterized by progressive loss of motor neurons. This is manifested in deterioration of strength, gait, speech, swallowing, respiratory function, and eventually…
Cellular Therapy for Spinal Muscular Atrophy: Pearls and Pitfalls
Abstract Spinal muscular atrophies (SMAs) include a series of neuromuscular genetic disorders due to mutations in the survival motor neuron 1 ( SMN1 ) gene. The SMA 5q form represents…
Clinical Trials to Date
Abstract Diseases that afflict the nervous system remain among the most difficult pathologies for clinicians to manage. Currently there are nearly 1000 clinical trials that have been launched worldwide to…
Gene Therapy for Amyotrophic Lateral Sclerosis: Therapeutic Transgenes
Abstract Amyotrophic lateral sclerosis (ALS) is a disease with a poorly understood etiology. There are a wide variety of abnormalities present, including mitochondrial dysfunction, glutamate excitotoxicity, oxidative stress, astrogliosis and…
Introduction to Gene and Stem-Cell Therapy
Abstract Motor neuron diseases, such as amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA), are devastating neurodegenerative diseases that are progressive and fatal. There is no cure and the…
