Hypomyelination with Atrophy of the Basal Ganglia and Cerebellum Market

Market Overview:

The 7 major hypomyelination with atrophy of the basal ganglia and cerebellum markets are expected to exhibit a growth rate (CAGR) of 2.20% during 2025-2035.

The hypomyelination with atrophy of the basal ganglia and cerebellum market has been comprehensively analyzed in IMARC's new report titled "Hypomyelination with Atrophy of the Basal Ganglia and Cerebellum Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2025-2035". Hypomyelination with atrophy of the basal ganglia and cerebellum (H-ABC) is an autosomal recessive disease caused by a failure in myelin development with severe impairments in both motor and cognitive functions. Mutations in the TUBB4A gene are the main cause, responsible for microtubule stability and neuronal function. The disorder typically presents early in childhood, and symptoms gradually increase over time. Patients usually present with developmental delay, spasticity, dystonia, ataxia, dysarthria, and cognitive regression. As the disease progresses, mobility and speech functions decline, necessitating supportive therapies to reduce symptoms. The diagnosis is generally made on genetic testing and MRI imaging, demonstrating characteristic hypomyelination and basal ganglia and cerebellar atrophy. Because H-ABC is rare and of low awareness, it is very often misdiagnosed, and appropriate treatments and interventions are delayed. Early and correct diagnosis is still essential to maximize patient care and possible treatment results.

The rising incidence of pathogenic TUBB4A gene mutations, which interfere with critical neuronal microtubule dynamics, is a key driver of the H-ABC market. Progress in genetic screening and neuroimaging technologies has enhanced early detection, resulting in higher diagnosis rates and better awareness among physicians. Moreover, expanding knowledge of disease pathophysiology has driven research into novel treatment strategies, such as gene therapy, neuroprotection, and myelin repair mechanisms. Supportive care including botulinum toxin for dystonia, baclofen for spasticity, and physical therapy for motor function gain is being used more frequently to control disease symptoms and increase quality of life. In addition, the increased launch of research programs aimed at oligodendrocyte-directed therapies and new neurodevelopmental treatments is providing enormous opportunities for therapeutic development. Although such advances have occurred, they continue to face issues such as few clinical trials, expensive treatment, and no disease-modifying agents. Greater stronger clinical trials, larger patient registries, and global partnerships are essential in the face of overcoming these issues to speed the pace of meaningful therapy development.

IMARC Group's new report provides an exhaustive analysis of the hypomyelination with atrophy of the basal ganglia and cerebellum market in the United States, EU4 (Germany, Spain, Italy, and France), United Kingdom, and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario, unmet medical needs, etc., have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the hypomyelination with atrophy of the basal ganglia and cerebellum market in any manner.

Recent Developments:

• SynaptixBio received Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) in March 2024 for their therapy intended for H-ABC. This designation grants benefits such as tax credits for qualifying clinical trials, a waiver of some regulatory fees, and market exclusivity at approval.
• A case study in July 2023 detailed the use of DBS to an adolescent patient with H-ABC caused by a TUBB4A mutation. The patient demonstrated significant improvement in motor function and dystonia following a staged DBS treatment, showing that DBS has therapeutic benefits for H-ABC patients, particularly in controlling movement disorders with the disorder.
• In April 2023, Oxfordshire biotech company SynaptixBio raised USD 16.6 million to push the world's first treatment for TUBB4A-related leukodystrophy, of which H-ABC is included. This fund will be used to finance current research and help trigger the commencement of human clinical trials.

Key Highlights:

• H-ABC affects fewer than 1 in 1,000,000 persons, making it extremely uncommon. There have been about 20 cases documented in the medical literature.
• The prognosis of H-ABC is variable. Some patients live into adulthood, with reports up to age 29. Yet, many patients have a progressive course, resulting in death in childhood or early adulthood.
• The symptoms typically appear in infancy or early childhood, typically within the first three years of life, however the majority of patients have a progressive course that results in mortality in youth or early adulthood.
• De novo autosomal dominant mutations in the TUBB4A gene, which codes for tubulin β-4A, a protein essential to the function of neuronal cell microtubules, have been connected to H-ABC.
• Progressive neurological impairments, stiffness, choreoathetosis, spasticity, dystonia, cerebellar ataxia, moderate to severe mental degeneration, and speech issues such as dysarthria or anarthria are characteristics of the disorder.

Time Period of the Study

• Base Year: 2024
• Historical Period: 2019-2024
• Market Forecast: 2025-2035

Countries Covered

• United States
• Germany
• France
• United Kingdom
• Italy
• Spain
• Japan

Analysis Covered Across Each Country

• Historical, current, and future epidemiology scenario
• Historical, current, and future performance of the hypomyelination with atrophy of the basal ganglia and cerebellum market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the hypomyelination with atrophy of the basal ganglia and cerebellum market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current hypomyelination with atrophy of the basal ganglia and cerebellum marketed drugs and late-stage pipeline drugs.

In-Market Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance

Late-Stage Pipeline Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:

Market Insights

• How has the hypomyelination with atrophy of the basal ganglia and cerebellum market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2024 and how are they expected to perform till 2035?
• What was the country-wise size of the hypomyelination with atrophy of the basal ganglia and cerebellum market across the seven major markets in 2024 and what will it look like in 2035?
• What is the growth rate of the hypomyelination with atrophy of the basal ganglia and cerebellum market across the seven major markets and what will be the expected growth over the next ten years?
• What are the key unmet needs in the market?

Epidemiology Insights

• What is the number of prevalent cases (​2019-2035​) of hypomyelination with atrophy of the basal ganglia and cerebellum across the seven major markets?
• What is the number of prevalent cases (​2019-2035​) of hypomyelination with atrophy of the basal ganglia and cerebellum by age across the seven major markets?
• What is the number of prevalent cases (​2019-2035) of hypomyelination with atrophy of the basal ganglia and cerebellum by gender across the seven major markets?
• How many patients are diagnosed (​2019-2035) with hypomyelination with atrophy of the basal ganglia and cerebellum across the seven major markets?
• What is the size of the hypomyelination with atrophy of the basal ganglia and cerebellum patient pool (2019-2024) across the seven major markets?
• What would be the forecasted patient pool (2025-2035) across the seven major markets?
• What are the key factors driving the epidemiological trend of hypomyelination with atrophy of the basal ganglia and cerebellum?
• What will be the growth rate of patients across the seven major markets?

Hypomyelination with Atrophy of the Basal Ganglia and Cerebellum: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

• What are the current marketed drugs and what are their market performance?
• What are the key pipeline drugs and how are they expected to perform in the coming years?
• How safe are the current marketed drugs and what are their efficacies?
• How safe are the late-stage pipeline drugs and what are their efficacies?
• What are the current treatment guidelines for hypomyelination with atrophy of the basal ganglia and cerebellum drugs across the seven major markets?
• Who are the key companies in the market and what are their market shares?
• What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the hypomyelination with atrophy of the basal ganglia and cerebellum market?
• What are the key regulatory events related to the hypomyelination with atrophy of the basal ganglia and cerebellum market?
• What is the structure of clinical trial landscape by status related to the hypomyelination with atrophy of the basal ganglia and cerebellum market?
• What is the structure of clinical trial landscape by phase related to the hypomyelination with atrophy of the basal ganglia and cerebellum market?
• What is the structure of clinical trial landscape by route of administration related to the hypomyelination with atrophy of the basal ganglia and cerebellum market?