Adrenoleukodystrophy Treatment Market: Introduction
- A rare genetic condition that causes the buildup of long chain fatty acids (VLCFAs) in the brain is known as adrenoleukodystrophy (ALD). A mutation in the ABCD1 gene on the X chromosome leads to ALD. Childhood cerebral ALD, Adrenomyelopathy, and Addison’s disease are the three types of adrenoleukodystrophy. Childhood cerebral ALD progresses very rapidly in children between the ages of 3 and 10 and causes severe disability and even death. Addison’s disease is caused when the adrenal glands are unable to produce enough hormones. It is also known as adrenal insufficiency. Adrenomyelopathy primarily affects adult men.
Key Drivers, Restrains and Opportunities of Global Adrenoleukodystrophy Treatment Market
- Rising awareness about rare diseases leads to increase in potential in research treatment. Moreover, rise in support and funds for research into new ALD treatments contribute to drive the market.
- Surge in number of patient is likely augment the market. For instance, the prevalence of adrenoleukodystrophy, globally, is 1 in 20,000 to 50,000 individuals as per the U.S. National Library of Medicine. 1 in every 21,000 men and 1 in 16,800 women thought to be affected by ALD in the U.S.
- Key industry players are engaged in the development of new products by investment in research & developing owing to a rise in the demand for products. This, in turn, is estimated to drive the market. For instance, Viking Therapeutics conducts a clinical trial program for novel VK0214. In September 2020, Viking Therapeutics launched phase I clinical trial testing with increasing doses of VK0214 to treat adrenoleukodystrophy (ALD). In July 2018, NeuroVia, Inc. initiated the Phase 1/2 clinical study of NV1205, in patients with childhood cerebral adrenoleukodystrophy (CCALD).
- However side-effects associated with stem cell therapy and corticosteroid replacement therapy is expected to hamper the market
- Government initiatives in developing countries to improve healthcare infrastructure leads to an increase the patient access and thereby offers significant opportunity in the market. Moreover, investment by key players to address the unmet medical needs is providing considerable opportunity in the market
North America to Capture Major Share of Global Adrenoleukodystrophy Treatment Market
- North America is expected to account for a major share of the global adrenoleukodystrophy treatment market. It is expected to maintain its dominance during the forecast period. Well-established healthcare infrastructure and presence of key market players are major factors that are anticipated to drive the market.
- The adrenoleukodystrophy treatment market in Asia Pacific is estimated to hold significant share of the market. The market in Asia Pacific is projected to expand at a rapid pace during the forecast period owing to a rise in awareness about rare diseases.
Key Players Operating in Global Adrenoleukodystrophy Treatment Market
The global adrenoleukodystrophy market is highly concentrated due to the presence of key players. A large number of manufacturers hold major share in their respective regions. Demand for adrenoleukodystrophy treatment products has increased in emerging as well as developed markets owing to a rise in prevalence of adrenoleukodystrophy. Growth strategies adopted by leading players are likely to drive the global market.
Major players operating in the global adrenoleukodystrophy treatment market are:
- agtc (Applied Genetic Technologies Corporation)
- Viking Therapeutics, Inc
- bluebird bio, Inc.
- SwanBio Therapeutics, Inc.
- MedDay Pharmaceuticals
- NeuroVia, Inc
Global Adrenoleukodystrophy Treatment Market: Research Scope
Global Adrenoleukodystrophy Treatment Market, by Therapy
- Stem cell transplant
- Gene therapy
- Corticosteroid replacement therapy
Global Adrenoleukodystrophy Treatment Market, by Types
- Childhood cerebral ALD
- Addison’s disease
This study by TMR is all-encompassing framework of the dynamics of the market. It mainly comprises critical assessment of consumers' or customers' journeys, current and emerging avenues, and strategic framework to enable CXOs take effective decisions.
Our key underpinning is the 4-Quadrant Framework EIRS that offers detailed visualization of four elements:
- Customer Experience Maps
- Insights and Tools based on data-driven research
- Actionable Results to meet all the business priorities
- Strategic Frameworks to boost the growth journey
The study strives to evaluate the current and future growth prospects, untapped avenues, factors shaping their revenue potential, and demand and consumption patterns in the global market by breaking it into region-wise assessment.
The following regional segments are covered comprehensively:
- North America
- Asia Pacific
- Latin America
- The Middle East and Africa
The EIRS quadrant framework in the report sums up our wide spectrum of data-driven research and advisory for CXOs to help them make better decisions for their businesses and stay as leaders.
Below is a snapshot of these quadrants.
1. Customer Experience Map
The study offers an in-depth assessment of various customers’ journeys pertinent to the market and its segments. It offers various customer impressions about the products and service use. The analysis takes a closer look at their pain points and fears across various customer touchpoints. The consultation and business intelligence solutions will help interested stakeholders, including CXOs, define customer experience maps tailored to their needs. This will help them aim at boosting customer engagement with their brands.
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The various insights in the study are based on elaborate cycles of primary and secondary research the analysts engage with during the course of research. The analysts and expert advisors at TMR adopt industry-wide, quantitative customer insights tools and market projection methodologies to arrive at results, which makes them reliable. The study not just offers estimations and projections, but also an uncluttered evaluation of these figures on the market dynamics. These insights merge data-driven research framework with qualitative consultations for business owners, CXOs, policy makers, and investors. The insights will also help their customers overcome their fears.
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The findings presented in this study by TMR are an indispensable guide for meeting all business priorities, including mission-critical ones. The results when implemented have shown tangible benefits to business stakeholders and industry entities to boost their performance. The results are tailored to fit the individual strategic framework. The study also illustrates some of the recent case studies on solving various problems by companies they faced in their consolidation journey.
4. Strategic Frameworks
The study equips businesses and anyone interested in the market to frame broad strategic frameworks. This has become more important than ever, given the current uncertainty due to COVID-19. The study deliberates on consultations to overcome various such past disruptions and foresees new ones to boost the preparedness. The frameworks help businesses plan their strategic alignments for recovery from such disruptive trends. Further, analysts at TMR helps you break down the complex scenario and bring resiliency in uncertain times.
The report sheds light on various aspects and answers pertinent questions on the market. Some of the important ones are:
1. What can be the best investment choices for venturing into new product and service lines?
2. What value propositions should businesses aim at while making new research and development funding?
3. Which regulations will be most helpful for stakeholders to boost their supply chain network?
4. Which regions might see the demand maturing in certain segments in near future?
5. What are the some of the best cost optimization strategies with vendors that some well-entrenched players have gained success with?
6. Which are the key perspectives that the C-suite are leveraging to move businesses to new growth trajectory?
7. Which government regulations might challenge the status of key regional markets?
8. How will the emerging political and economic scenario affect opportunities in key growth areas?
9. What are some of the value-grab opportunities in various segments?
10. What will be the barrier to entry for new players in the market?
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