Adrenoleukodystrophy, Or Ald, Is A Deadly Genetic Disease That Affects 1 In 18,000 People
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: https://univdatos.com/report/global-adrenoleukodystrophy-pipeline-analysis-2019
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.
Adrenoleukodystrophy Pipeline Analysis
Products covered by Phase
- Phase III
- Phase II
- Phase I
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
- SOM Biotech
- Viking Therapeutics