Adrenoleukodystrophy Pipeline Overview
Adrenoleukodystrophy is a fatal progressive neurogenerative disorder affecting brain white matter. The common form of ALD is X-linked (X-ALD) and results from mutation of ABCD1 encoded very long chain fatty acid (VLCFA) transporter. It is clinically heterogeneous with the cerebral form being the most severe. X-linked Adrenoleukodystrophy (X-ALD) is a rare X-linked metabolic disorder caused by variants in the ABCD1 gene which causes a deficiency in adrenoleukodystrophy protein (ALDP) and subsequent accumulation of very long chain fatty acids (VLCFA).
The symptoms of Adrenoleukodystrophy are highly variable. There are mainly three types which are present in approximately 90-95% of the affected people: a childhood cerebral form; an adrenomyeloneuropathy type; and an adrenal insufficiency only type.
The diagnosis of ALD, therefore, can be made with the highly sensitive and specific plasma VLCFA assay. When an abnormal pattern is detected with an identifiable ABCD1 mutation in a presymptomatic patient (or in the setting of classic clinical disease in others), males are determined to have the biochemical defect of ALD. Disease may manifest at any age. Most ALD males will develop primary adrenal insufficiency (AI) in childhood or adolescence, and most surviving males will develop spinal cord disease (adrenomyeloneuropathy [AMN]) during adulthood.
The therapeutic pipeline of Adrenoleukodystrophy consists of approximately 9+ products in different stages of development. Currently, 3+ drugs are in Phase III development and major drugs are in late stage.
Top Company Analysed
Some of the key players include Applied Genetic Technologies Corporation; Bluebird bio; Magenta Therapeutics; MedDay Pharmaceuticals; Minoryx Therapeutics; NeuroVia; Orpheris; ReceptoPharm; SOM Biotech; and Viking Therapeutics.
Scope of the report:
• Provides an overview of therapeutic pipeline activity for Adrenoleukodystrophy across the complete product development cycle including all clinical and non-clinical stages
• The report comprises of detailed profiles of Adrenoleukodystrophy therapeutic products with key coverage of developmental activities including licensing & collaboration deals, patents issued, designations, technologies and chemical information
Enquire before purchasing this report: https://www.sdki.jp/sample-request-111421
• Therapeutic assessment of the active pipeline products by stage, product type, molecule type, and route of administration
• Detailed profiles of the discontinued products have been included in the report
Reasons to buy:
• The Adrenoleukodystrophy pipeline presents the detailed profile of drugs. The analysis offered in the report is a combination of deep dive secondary research and input from Key Opinion Leader of the industry
• The report presents a quick review of the current scenario regarding the drug development of the indication at one glance
• The report covers in-depth analysis of prominent industry peers with a primary focus on company consolidation, designation, technology, agreements and patents regarding the therapy
• Detailed examination on diagnosis, treatment and guidelines prevailing in the industry
• Examination of industry attractiveness with the help of approval timelines
• The study comprehensively covers the market across drugs in different phases of development
• Extensive domain knowledge on therapy areas support the client in decision-making process regarding their therapeutic portfolio by identifying the reason behind the discontinuation of the products
Request to Fill The Form To get Sample Copy of This Report: https://www.sdki.jp/sample-request-111421
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.
Maritime Patrol Aircraft Market
Europe Inflight Entertainment and Connectivity Market
North America Inflight Entertainment and Connectivity Market
Asia-Pacific Ammunition Market
Helicopter Simulator Market
Inflight Entertainment and Connectivity Market
Military Aircraft Simulation and Training Market
Europe Aviation Market
Asia-Pacific Commercial Aircraft Cabin Interior Market
North America General Aviation Market
Asia-Pacific Small UAV Market
Military Rotorcraft Market