The Spectrum of Motor Neuron Diseases: From Childhood Spinal Muscular Atrophy to Adult Amyotrophic Lateral Sclerosis.
Reads0
Chats0
TLDR
This special issue offers a timely and comprehensive look into the recent research advances, current clinical considerations, and therapeutic prospects currently in development or being tested for motor neuron diseases.About:
This article is published in Neurotherapeutics.The article was published on 2015-04-01 and is currently open access. It has received 1 citations till now. The article focuses on the topics: Neuromuscular disease & Spinal muscular atrophy.read more
Citations
More filters
Journal ArticleDOI
The Interplay Between Neuroinfections, the Immune System and Neurological Disorders: A Focus on Africa
TL;DR: The interplay between neuroinfections, immune system, neuroinflammation, and neurological disorders is described, and how understanding this can be exploited for the development of novel diagnostics and therapeutics for improved patient care is described.
References
More filters
Journal ArticleDOI
TDP-43 Proteinopathy and ALS: Insights into Disease Mechanisms and Therapeutic Targets
TL;DR: It is demonstrated how diverse environmental stressors linked to stress granule formation, as well as mutations in genes encoding RNA processing proteins and protein degradation adaptors, initiate ALS pathogenesis via TDP-43.
Journal ArticleDOI
Protective and Toxic Neuroinflammation in Amyotrophic Lateral Sclerosis
TL;DR: This review will focus on the cellular players involved in neuroinflammation in ALS and current therapeutic strategies to enhance neuroprotection and suppress neurotoxicity with the goal of arresting the progressive and devastating nature of ALS.
Journal ArticleDOI
The Genetics of Spinal Muscular Atrophy: Progress and Challenges
TL;DR: This review incorporates an overview of the clinical manifestations and genetics of SMA, and describes recent advances in the understanding of mechanisms of disease pathogenesis and development of novel treatment strategies.
Journal ArticleDOI
Patient-Perceived Outcomes and Quality of Life in ALS.
TL;DR: A variety of outcome measures are used in clinical practice and in research to assess patients with amyotrophic lateral sclerosis (ALS), but there may be discordance between traditional outcome measures such as strength and physical function, and patient-perceived measures of well-being.
Journal ArticleDOI
Spinal Muscular Atrophy Therapeutics: Where do we Stand?
TL;DR: Some of the therapeutic strategies that have been developed to activate SMN2 expression, modulate splicing of theSMN2 pre-mRNA, or replace SMN1 by gene therapy are highlighted.