Adrenoleukodystrophy (ADL) is defined as a X-linked disorder which is more prominent in males. This disorder affects the adrenal glands. In this disease the fatty covering over the neurons and spinal cord starts deteriorating, thus reducing nerve’s ability to transfer the information. The damage to adrenal glands leads to reduction in the level of adrenocortical hormones. This reduced level in turn causes weakness, weight loss, coma, and changes in skin. Children with the cerebral form of X-linked adrenoleukodystrophy experience difficulty in reading, writing, understanding speech, and comprehending written material. Individuals with the cerebral form of X-linked adrenoleukodystrophy usually survive only a few years after symptoms begin. Symptoms of the adrenomyeloneuropathy occur between early adulthood and middle age.
Affected individuals develop progressive stiffness and weakness in their legs. This disease is very rare and has a low prevalence rate. Adrenal dysfunction may be treated with steroids (such as cortisol) if the adrenal gland is not producing enough hormones. Any specific treatment for X-linked adrenoleukodystrophy is not available. In May 2018, bluebird bio Inc.’s Lenti-D was granted breakthrough therapy designation for the treatment of patients with cerebral adrenoleukodystrophy (CALD), a rare, serious and life-threatening hereditary neurological disorder by the USFDA (Food and Drug Administration). Further, Viking Therapeutics and Orpheris Inc. are other key players in the pipeline.
The report covers disease overview along with currently available diagnostic and treatment methods; detailed product profiles of pipeline assets; and segmentation of pipeline assets by company, phase of development, route of administration (RoA), mechanism of action (MoA), and molecule type.
Highlights of the report:
Recent advancements in ADL pipeline:
Objective of the report:
Segmentation of phases featured in the report:
Coverage of marketed and pipeline research activities in the report:
Comprehensive product profiles of marketed and pipeline products covering their research and development activities; regulatory milestones of marketed products; and competitive landscape of other strategic development affairs outlining drug designations, collaboration, and licensing details, mergers and acquisitions, patent information, and technology used.
Epidemiology forecast of ADL in the seven major markets (7MM):
This section of the report provides incident/prevalent cases of ADL along with further segmentation of patient pool by age, gender, and severity (depending on the availability of information) in the seven major markets (7MM) i.e. the U.S., EU5 (France, Germany, Spain, Italy, and the U.K.), and Japan.
Recent and upcoming strategic development activities and therapeutic segmentation in the report:
This part of the report covers details about the key recent and upcoming national and international conferences in the year. Also provided is the segmentation of marketed therapeutic assets by region, pipeline therapeutic assets by clinical trial region and status, attribute analysis, and launch timeline analysis of higher phase products (Filed, Phase III).
Company Profiles in the report:
The report also provides detailed profiles of key companies covering their business overview and services offered by them.