DelveInsight’s “Adrenoleukodystrophy (ALD) Market Insights, Epidemiology, and Market Forecast 2034” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Adrenoleukodystrophy market size, share, trends, and growth opportunities in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).
The Adrenoleukodystrophy market report covers emerging drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2020 to 2034. It also evaluates the current treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Adrenoleukodystrophy Overview
Adrenoleukodystrophy (ALD) is a rare genetic disorder that affects the nervous system and adrenal glands. It is characterized by the progressive degeneration of the white matter of the brain and the dysfunction of the adrenal glands. Here is an in-depth look at ALD, including its causes, signs and symptoms, diagnosis, and treatment options.
Adrenoleukodystrophy is a type of leukodystrophy, which refers to a group of genetic disorders that involve the progressive degeneration of myelin—the protective covering of nerve cells. ALD primarily affects the white matter of the brain and the adrenal glands. It predominantly impacts males, though females can be carriers or, in rare cases, show symptoms.
The condition is linked to mutations in the ABCD1 gene located on the X chromosome. This gene is responsible for coding a protein called ALDP (adrenoleukodystrophy protein), which is crucial for the breakdown of very long-chain fatty acids (VLCFAs) in the peroxisomes, cellular organelles involved in lipid metabolism.
Causes
Genetic Mutation: ALD is inherited in an X-linked recessive pattern. This means that the condition is caused by mutations in the ABCD1 gene on the X chromosome. In males, who have one X and one Y chromosome, a mutation in the ABCD1 gene leads to the development of the disorder. In females, who have two X chromosomes, one mutated gene can lead to them being carriers.
Disruption of VLCFA Metabolism: The mutation impairs the function of ALDP, which leads to the accumulation of VLCFAs in the brain and adrenal glands. This accumulation is toxic and causes damage to myelin in the brain and adrenal cortex, leading to the symptoms of ALD.
Signs and Symptoms
1. Neurological Symptoms: – Childhood Form: The most common and severe form, known as cerebral ALD, typically begins in early childhood. Symptoms include behavioral changes, learning difficulties, and progressive loss of motor skills. As the condition advances, children may experience loss of vision, seizures, and loss of cognitive functions. – Adolescent/Adult Form: Symptoms may include progressive neurological decline, such as motor dysfunction, impaired cognitive functions, and psychiatric symptoms. Onset can occur in late adolescence or adulthood.
2. Adrenal Insufficiency: – Addison’s Disease: One of the key features of ALD is adrenal insufficiency, also known as Addison’s disease. Symptoms include fatigue, weight loss, low blood pressure, and darkened skin.
3. Behavioral and Cognitive Changes: – Children may exhibit behavioral problems, including hyperactivity, aggression, or withdrawal. – Cognitive decline can affect memory, attention, and learning abilities.
4. Physical Symptoms: – Difficulty with movement and coordination, including problems with walking and fine motor skills. – Loss of muscle strength and spasticity.
Diagnosis
1. Clinical Evaluation: – Medical History: A detailed medical and family history is crucial for diagnosis, especially considering the X-linked inheritance pattern. – Neurological Examination: Evaluates symptoms related to motor and cognitive functions.
2. Imaging Studies: – MRI (Magnetic Resonance Imaging): MRI of the brain can reveal characteristic patterns of white matter damage associated with ALD. In cerebral ALD, MRI shows extensive demyelination.
3. Laboratory Tests: – Plasma VLCFA Levels: Elevated levels of very long-chain fatty acids in the blood are indicative of ALD. This test is used to confirm the diagnosis and monitor disease progression.
4. Genetic Testing: – ABCD1 Gene Analysis: Genetic testing can identify mutations in the ABCD1 gene, confirming the diagnosis of ALD.
5. Adrenal Function Tests: – ACTH Stimulation Test: Assesses the functionality of the adrenal glands, helping to diagnose adrenal insufficiency.
Treatment
1. Hormone Replacement Therapy: – Adrenal Insufficiency Management: Lifelong treatment with corticosteroids (such as hydrocortisone) is required to manage adrenal insufficiency.
2. Disease-Modifying Treatments: – Hematopoietic Stem Cell Transplantation (HSCT): This treatment aims to halt the progression of neurological symptoms, especially in early stages of cerebral ALD. It involves replacing the patient’s stem cells with healthy ones from a donor. – Gene Therapy: Experimental approaches are being developed to correct the genetic defect in ALD. These therapies are still largely in the research phase but hold promise for future treatment.
3. Symptomatic Treatment: – Physical Therapy: Helps manage and improve motor skills and mobility. – Occupational Therapy: Assists in daily living activities and cognitive rehabilitation. – Speech Therapy: Addresses communication difficulties.
4. Supportive Care: – Psychological Support: Counseling and support groups can be beneficial for patients and families dealing with the emotional impact of the disease. – Educational Support: Special education services can help children with learning difficulties resulting from neurological damage.
Future Directions
Research into adrenoleukodystrophy is ongoing, with a focus on improving early diagnosis, developing effective treatments, and exploring potential gene therapies. Advances in molecular genetics and stem cell research hold the potential to transform the management of this debilitating condition.
Adrenoleukodystrophy is a severe genetic disorder that impacts both neurological and endocrine systems. Early diagnosis and intervention are critical for managing the condition and improving quality of life. Advances in treatment options, including stem cell therapy and gene therapy, offer hope for better management and potential cures in the future. Continued research and support are essential for improving outcomes for individuals affected by ALD.
Adrenoleukodystrophy (ALD) Market Key Facts
- In 2023, the United States accounted for the largest market size (~USD 400 Million) of adrenoleukodystrophy, in comparison to EU4 (Germany, Spain, Italy, France), the United Kingdom, and Japan.
- Adrenoleukodystrophy (ALD) is an X-linked disorder resulting from a defect in peroxisomal beta-oxidation of very-long-chain fatty acids (VLCFA).
- Based on the age of onset and severity of its symptoms, ALD is classified into three main types: childhood cerebral adrenoleukodystrophy (CALD), adrenomyeloneuropathy (AMN), and Addison’s disease.
- According to DelveInsight’s estimates, CALD accounted for the largest share (~50%) of diagnosed ALD cases in the United States in 2023.
- Adrenoleukodystrophy (ALD) is a rare genetic disorder that predominantly affects male during childhood. In 2023, around 60% of ALD cases were diagnosed in male based on the gender-specific prevalence of the disease in the 7MM.
- At present, there is no cure for ALD. One of the effective treatment option for cerebral ALD is a stem cell transplant, a procedure in which the patient receives blood stem cells from a genetically matched donor. Hematopoietic stem cell therapy is effective in X-ALD if initiated early, but treatment for X-AMN is lacking
- The market size of adrenoleukodystrophy (ALD) in the 7MM is expected to increase due to increasing awareness of newborn screening programs, growing number of cases of cerebral adrenoleukodystrophy (CALD), and expected approval of potential therapies over the forecast period (2024-2034).
- In September 2022, Bluebird Bio received the US FDA accelerated approval for SKYSONA gene therapy for early, active cerebral adrenoleukodystrophy (CALD).
- Several emerging therapies like Leriglitazone (MIN-102), PXL770, and others are in development for ALD to address the existing unmet medical needs in the market.
- In January 2024, Minoryx Therapeutics announced that the EMA’s Committee for Medicinal Products for Human Use (CHMP) has recommended not to grant marketing authorization for Nezglyal (leriglitazone) as a treatment of X-ALD.
Adrenoleukodystrophy (ALD) Market
The market outlook section of the report helps to build a detailed comprehension of the historical, current and forecasted Adrenoleukodystrophy market size by analyzing the impact of current and emerging pipeline therapies. It also thoroughly assesses the market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.
The report gives complete detail of the Adrenoleukodystrophy market trend for each marketed drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competition with other therapies, brand value, and their impact on the market.
Adrenoleukodystrophy Epidemiology Assessment
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pool, future trends, and views of key opinion leaders.
The Report Covers the Adrenoleukodystrophy Epidemiology, Segmented as –
Total Prevalent Population of ALD in the 7MM [2020–2034]
Gender-specific Prevalence of ALD in the 7MM [2020–2034]
Gender-specific Diagnosed Prevalent Population of ALD in the 7MM [2020–2034]
Type-specific Diagnosed Prevalence of ALD in males in the 7MM [2020–2034]
Adrenoleukodystrophy Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the uptake rate of potential drugs recently launched in the Adrenoleukodystrophy market or expected to be launched during the study period. The analysis covers the Adrenoleukodystrophy market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.
The report also covers the Adrenoleukodystrophy pipeline development activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyses recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
Learn How the Adrenoleukodystrophy Market Will Evolve and Grow by 2034 @ https://www.delveinsight.com/sample-request/adrenoleukodystrophy-market
Adrenoleukodystrophy (ALD) Therapeutics Analysis
Several major pharma and biotech companies are developing therapies for Adrenoleukodystrophy (ALD). Currently, bluebird bio is leading the therapeutics market with its Adrenoleukodystrophy (ALD) drug candidates in the most advanced stage of clinical development.
Leading Companies in the Adrenoleukodystrophy (ALD) Therapeutics Market Include:
bluebird bio
Applied Genetic Technologies Corporation
Poxel
Minoryx Therapeutics
Orpheris
MedDay Pharmaceuticals
Viking Therapeutics
bluebird bio
Minoryx Therapeutics, S.L.
MedDay Pharmaceuticals
Magenta Therapeutics
Orpheris Inc.
SOM Biotech
And Many Others
Adrenoleukodystrophy (ALD) Emerging and Marketed Drugs Covered in the Report Include:
Elivaldogene autotemcel: bluebird bio
Leriglitazone: Minoryx Therapeutics
OP 101: Ashvattha Therapeutics
MGTA-456: Magenta Therapeutics, Inc.
VK 0214: Viking Therapeutics
Adrenoleukodystrophy gene therapy (Research Programme) – AGTC
And Many More
The Report Covers the In-depth Assessment of the Emerging Drugs & Key Companies. Download the Sample Report to Learn More @ https://www.delveinsight.com/sample-request/adrenoleukodystrophy-market
Table of Content
1. Key Insights
2. Executive Summary
3. Adrenoleukodystrophy Competitive Intelligence Analysis
4. Adrenoleukodystrophy Market Overview at a Glance
5. Adrenoleukodystrophy Disease Background and Overview
6. Adrenoleukodystrophy Patient Journey
7. Adrenoleukodystrophy Epidemiology and Patient Population (In the US, EU5, and Japan)
8. Adrenoleukodystrophy Treatment Algorithm, Current Treatment, and Medical Practices
9. Adrenoleukodystrophy Unmet Needs
10. Key Endpoints of Adrenoleukodystrophy Treatment
11. Adrenoleukodystrophy Marketed Products
12. Adrenoleukodystrophy Emerging Drugs and Latest Therapeutic Advances
13. Adrenoleukodystrophy Seven Major Market Analysis
14. Attribute Analysis
15. Adrenoleukodystrophy Market Outlook (In US, EU5, and Japan)
16. Adrenoleukodystrophy Access and Reimbursement Overview
17. KOL Views on the Adrenoleukodystrophy Market
18. Adrenoleukodystrophy Market Drivers
19. Adrenoleukodystrophy Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
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