Giant Axonal Neuropathy Pipeline Insights

DelveInsight’s “Giant Axonal Neuropathy Pipeline Insight” report provides comprehensive insights about key companies and pipeline drugs in the Giant Axonal Neuropathy pipeline landscapes. 

The report comprises Giant Axonal Neuropathy pipeline drug profiles, including clinical and non-clinical stage products. It also includes the Giant Axonal Neuropathy therapeutics assessment by product type, stage, route of administration, and molecule type and further highlights the inactive Giant Axonal Neuropathy pipeline products.     

Giant Axonal Neuropathy Overview

Giant axonal neuropathy (GAN) is a rare inherited genetic disorder that severely affects the peripheral as well as the central nervous system. GAN is characterized by abnormally large and dysfunctional axons called giant axons. The onset of GAN ranges from early infancy to late childhood

This neurodegenerative disorder is caused by mutations in the GAN gene located on chromosome 16 at 16q24.1 which provides instructions for making a protein called gigaxonin protein. The nonstandard gigaxonin protein causes a portion of the nerve cell called the axon to swell up with deposits of tiny threads of a protein called neurofilaments, giving the presence of giant axons. The giant axons cause degeneration and abnormal functioning of the peripheral nervous system. This condition is inherited in an autosomal recessive pattern, which means both copies of the gene present in each cell have mutations. The parents of an individual with an autosomal recessive condition each carry one copy of the mutated gene, but they usually do not show signs and symptoms of the condition. As per some studies, since the first GAN report in 1972, more than 50 GAN gene mutations have been identified.

Some of the key takeaways from the Giant Axonal Neuropathy Pipeline Report:

• Companies across the globe are diligently working toward the development of novel treatment therapies with a considerable amount of success over the years. Key players, such as Taysha Gene Therapies, etc., are developing therapies for the treatment of Giant Axonal Neuropathy.
• Emerging therapies such as TSHA-120, are expected to have a significant impact on the  Giant Axonal Neuropathy market in the coming years.

Get an overview of pipeline landscape @ Giant Axonal Neuropathy Clinical Trials Analysis 

Giant Axonal Neuropathy Pipeline Therapies along with Key Players:

• TSHA-120: Taysha Gene Therapies

Scope of Giant Axonal Neuropathy Pipeline Drug Insight    

• Coverage: Global 
• Major Players: Taysha Gene Therapies, and others.
• Pipeline Therapies: TSHA-120, and others.

Table of Contents

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