Adrenoleukodystrophy is a rare genetic disorder that affects one in 17,000 people around the world. It is more common in males than in females, and the disease usually manifests in the early adolescence and early adulthood. While males are more likely to be affected by the disorder, female carriers account for twenty to forty percent of all cases. There is currently no cure for this disease, but research is ongoing.
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The ABCD1 gene codes for a protein called ALD, or adrenoleukodystrophy. This protein is necessary for normal fat metabolism in the brain and spinal cord. Without this protein, very long chain fatty acids are not broken down, and accumulate in the tissues. In severe cases, the brain and adrenal cortex may become damaged, resulting in the onset of symptoms like seizures, muscle weakness, and blindness. Children are most likely to develop the most severe form of adrenoleukodystrophy, which is also called childhood adrenoleukodystrophic disease (ALD). Usually diagnosed in childhood, this disorder causes visual and/or behavioral problems. It may even progress to the point where the affected person becomes blind. There are also several rare forms of adrenoleukodytrophy – Pelizaeus-Merzbacher syndrome, and X-linked adrenoleukodystic dystrophy.
Treatment used for Adrenoleukodystrophy (ALD) are stem cell transplant, adrenal insufficiency treatment, gene therapy and dietary therapy.
Global Adrenoleukodystrophy Drugs Market: Drivers
Growing prevalence of rare disorder and increasing investment for developing novel drug to treat rare disorder is driving Adrenoleukodystrophy drug market growth. For instance, in May 2020, SwanBio Therapeutics, a developer of biological therapeutics, announced that they have raised US$ 52 million for advancement of their viral base gene therapy for adrenomyeloneuropathy. Funds were raised with the help of series A financing round.
Global Adrenoleukodystrophy Drugs Market: COVID-19 Impact Analysis
COVID-19 has affected several markets around the world. In case of global adrenoleukodystrophy drugs market, adrenoleukodystrophy patients are facing challenges due to reallocation of neurological intensive beds and neurological wards to COVID-19 patients. As a result, there is difficulty in management of emergencies related to neurological disorders. Furthermore, implementation of lockdowns and restrictions on transportation by governments of various countries have affected the supply chain activities, and led to a shortage in inventories of pharmaceutical companies.
Global Adrenoleukodystrophy Drugs Market: Restraints
Side effects such as interstitial pneumonia, liver damage and mucositis caused due to adoption of stem cell treatment for adrenoleukodystrophy is expected to hinder market growth. Additionally, in adrenal insufficiency treatment, hydrocortisone is a key drug which affects cortisol levels in adrenoleukodystrophy patients and also weakens the immune system. Moreover, gene therapy may lead to mutation of cells and inflammation because of wrong incorporation of DNA on DNA locations is again expected to restrict global adrenoleukodystrophy drugs market growth.
Global Adrenoleukodystrophy Drugs Market Regional Analysis
On basis of regions, the global Adrenoleukodystrophy market is segmented into North America, Latin America, Europe, Asia Pacific, Middle East, and Africa. In North America, pharmaceutical companies are focusing on advancement of therapies for treatment of adrenoleukodystrophy. For instance, in 2019, Magneta Therapeutics received approval for MGTA-456 regenerative medicine advance therapy designation for treatment of adrenoleukodystrophy from the U.S Food Drug and Administration.
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In 2018, Europe PMC claimed that adrenal insufficiency is a life threating condition with mortality rate of 0.5 in 100 patients per year. Moreover, pharmaceutical companies are focusing on research and development of treatments for adrenoleukodystrophy to strengthen their share in the global adrenoleukodystrophy drugs market. For instance, in January 2020, Minoryx Therapeutics, a company specialised in innovation of drugs for central nervous system conditions, received an approval for lead drug candidate, leriglitazone (MIN-102), for treatment of all forms of x- adrenoleukodystrophy.
Global Adrenoleukodystrophy Drugs Market: Key Players
Key operating players in global adrenoleukodystrophy drugs market are Applied Genetic Technologies Corporation, MedDay SA, Bluebird bio, Inc., Minoryx Therapeutics S.L, ReceptoPharm Inc., Pfizer Inc., SOM Innovation Biotech, S.A, SwanBio Therapeutics and Viking Therapeutics, Inc.
Market Taxonomy:
On the Basis of Treatment, the global adrenoleukodystrophy drugs market is segmented into:
- Adrenal Insufficiency Treatment
- Gene Therapy Drugs
- Dietary Supplements
On the Basis of Patient Type, the global adrenoleukodystrophy drugs market is segmented into:
- Adult
- Pediatrics
On the Basis of Distribution Channel, the global adrenoleukodystrophy drugs market is segmented into:
- Hospital Pharmacies
- Retail Pharmacies
- Online Pharmacies
On the Basis of Indication, the Global Adrenoleukodystrophy Drugs Market is segmented into:
- Childhood Adrenoleukodystrophy
- Addison Disease
- Adrenomyeloneuropathy
On the basis of region, the global adrenoleukodystrophy drugs market is segmented into:
- North America
- Latin America
- Europe
- Asia Pacific
- Middle East
- Africa
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Table of Content
Chapter 1 Industry Overview
1.1 Definition
1.2 Assumptions
1.3 Research Scope
1.4 Market Analysis by Regions
1.5 Adrenoleukodystrophy Drugs Market Size Analysis from 2021 to 2028
11.6 COVID-19 Outbreak: Adrenoleukodystrophy Drugs Industry Impact
Chapter 2 Global Adrenoleukodystrophy Drugs Competition by Types, Applications, and Top Regions and Countries
2.1 Global Adrenoleukodystrophy Drugs (Volume and Value) by Type
2.3 Global Adrenoleukodystrophy Drugs (Volume and Value) by Regions
Chapter 3 Production Market Analysis
3.1 Global Production Market Analysis
3.2 Regional Production Market Analysis
Chapter 4 Global Adrenoleukodystrophy Drugs Sales, Consumption, Export, Import by Regions (2016-2021)
Chapter 5 North America Adrenoleukodystrophy Drugs Market Analysis
Chapter 6 East Asia Adrenoleukodystrophy Drugs Market Analysis
Chapter 7 Europe Adrenoleukodystrophy Drugs Market Analysis
Chapter 8 South Asia Adrenoleukodystrophy Drugs Market Analysis
Chapter 9 Southeast Asia Adrenoleukodystrophy Drugs Market Analysis
Chapter 10 Middle East Adrenoleukodystrophy Drugs Market Analysis
Chapter 11 Africa Adrenoleukodystrophy Drugs Market Analysis
Chapter 12 Oceania Adrenoleukodystrophy Drugs Market Analysis
Chapter 13 South America Adrenoleukodystrophy Drugs Market Analysis
Chapter 14 Company Profiles and Key Figures in Adrenoleukodystrophy Drugs Business
Chapter 15 Global Adrenoleukodystrophy Drugs Market Forecast (2021-2027)
Chapter 16 Conclusions
Research Methodology
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