Adrenoleukodystrophy Market — Industry Analysis, Size, Share, Growth, Trends, and Forecast 2019–2025

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Adrenoleukodystrophy

Adrenoleukodystrophy is an X-linked disorder which results from a defect in peroxisomal beta oxidation of very long chain fatty acids (VLCFA). The presence of VLCFA in myelin induces myelin instability, which results in an immune mediated process in which the presentation of a lipid antigen may result in substantial myelin destruction.

The most common form of ALD is X-linked and results from mutation of the ABCD1 encoded very long chain fatty acid transporter. It is clinically heterogenous with the cerebral form of being the most severe. It is diagnosed usually in boys between 4 and 8 years. The symptoms progress rapidly through declines in cognition, learning and behavior to paralysis and ultimately to a vegetative state and death. The spectrum of clinical features associated with ALDP mutation is broad, ranging from adrenocortical insufficiency to slowly progressive myelopathy to cerebral demyelination.

For detailed analysis of the TOC for the reports browse through: Adrenoleukodystrophy Market

There are only two available treatments for childhood cerebral X-ALD: Lorenzo’s Oil (a dietary supplement) and stem cell transplantation. The former does not significantly improve clinical symptoms of the disease, while the latter has serious limitations. The current therapeutic option for the most severe ALD cases is bone marrow transplant; however, the procedure itself carries substantial risk and is not always successful. The treatment options for children at more advanced stages of X-ALD or the adult form of the disease are very limited.

Some of the key players involved in the development of the therapeutics for the treatment of Adrenoleukodystrophy include Minoryx Therapeutics; Bluebird bio; MedDay Pharmaceuticals; ReceptoPharm; Magenta Therapeutics; Orpheris; SOM Biotech; Applied Genetic Technologies Corporation and Viking Therapeutics among others.

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Adrenoleukodystrophy Pipeline Analysis

Report Segmentation

Products covered by Phase

Phase III

Phase II

Phase I

Pre-clinical

Discontinued

Therapeutic Assessment by Stages

Product Type

Route of Administration

Molecule Type

Top Companies Analyzed

Applied Genetic Technologies Corporation

Bluebird bio

Magenta Therapeutics

MedDay Pharmaceuticals

Minoryx Therapeutics

NeuroVia

Orpheris

ReceptoPharm

SOM Biotech

Viking Therapeutics

The Adrenoleukodystrophy pipeline analysis report can be customized to the country level or any other competitive segment. Besides this, UMI understands that you may have your own business need, hence we also provide fully customized solutions to clients.

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