Global Adrenoleukodystrophy treatment – Trends and Highlights / Applied Genetic Technologies Corporation; Bluebird bio; Magenta Therapeutics.

Adrenoleukodystrophy (ALD) is a rare genetic condition that causes the buildup of very-long-chain fatty acids (VLCFAs) in the brain. The defective gene in ALD, commonly referred to as a genetic mutation, can cause several different but related conditions: adrenomyelopathy (AMN), Addison’s disease, and - the most common and most devastating form - cerebral ALD. Cerebral ALD strikes boys between ages 4 and 10, leading to permanent disability and death usually within four to eight years.

For detailed analysis on the Global Adrenoleukodystrophy Pipeline Analysis browse through https://univdatos.com/report/global-adrenoleukodystrophy-pipeline-analysis-2019

Signs and symptoms of the adrenomyeloneuropathy type appear between early adulthood and middle age. Affected individuals develop progressive stiffness and weakness in their legs (paraparesis), experience urinary and genital tract disorders, and often show changes in behavior and thinking ability. Most people with the adrenomyeloneuropathy type also have adrenocortical insufficiency. In some severely affected individuals, damage to the brain and nervous system can lead to an early death.

For detailed analysis on the symptoms of Global Adrenoleukodystrophy Pipeline Analysis browse through https://univdatos.com/report/global-adrenoleukodystrophy-pipeline-analysis-2019

The diagnosis of the disease includes genetic counseling, differential diagnosis, biochemical and molecular diagnosis. Genetic counseling must be offered to the parents of affected boys, adult males, and women with X-ALD and their family to detect: carriers who can be offered prenatal diagnosis, and asymptomatic or pre-symptomatic men or women who can benefit from therapeutic interventions. Regular follow-up in presymptomatic males can prevent serious morbidity and mortality.

For detailed analysis on the diagnosis in Global Adrenoleukodystrophy Pipeline Analysis browse through https://univdatos.com/report/global-adrenoleukodystrophy-pipeline-analysis-2019

Despite significant mortality risk, allogeneic HCT remains the only therapeutic intervention that can arrest the progression of cerebral demyelination in X-ALD, provided the procedure is performed very early, i.e., when affected boys or men have no or minor symptoms due to cerebral demyelinating disease. In the future, transplantation of autologous hematopoietic stem cells that have been genetically corrected with a lentiviral vector before re-infusion might become an alternative to autologous HCT, once the very encouraging results obtained in the first two treated patients would have been extended to a larger number of patients with cerebral X-ALD.

For detailed analysis on the management in Global Adrenoleukodystrophy Pipeline Analysis browse through https://univdatos.com/report/global-adrenoleukodystrophy-pipeline-analysis-2019

There are currently only two available treatments for childhood cerebral ALD: Lorenzo's oil and stem cell transplantation, using either umbilical cord stem cells or bone marrow stem cells. Both treatment approaches have shown promise and been effective for some boys with ALD, but they also both have drawbacks. 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 the late stage.

Request for Sample of the report browse through https://univdatos.com/report/global-adrenoleukodystrophy-pipeline-analysis-2019

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. Several M&A’s along with partnerships have been undertaken by these players to facilitate costumers with hi-tech and innovative products.

Top Company Profiles
• Applied Genetic Technologies Corporation
• Bluebird bio
• Magenta Therapeutics
• MedDay Pharmaceuticals
• Minoryx Therapeutics
• NeuroVia
• Orpheris
• ReceptoPharm
• SOM Biotech
• Viking Therapeutics

CONTACT US
E-mail: sales@univdatos.com
Phone: +91 783860491
Address-: 4th & 5th Floor,
C80B, Sector 8, Noida,
Pin code- 201301, UP, India

Aout Us
UnivDatos Market Insights (UMI), is a passionate market research firm and a subsidiary of Universal Data Solutions. We believe in delivering insights through Market Intelligence Reports, Customized Business Research, and Primary Research. Our research studies are spread across topics across the world, we cover markets over 100 countries using smart research techniques and agile methodologies. We offer in-depth studies, detailed analysis, and customized reports that help shape winning business strategies for our clients

This release was published on openPR.