Menkes disease, also known as kinky hair disease, is a rare genetic disorder characterized by abnormalities in copper transport. It results in reduced levels of copper in the body and affects many parts of the body including the brain and nervous system. The current treatments available aim to manage symptoms and improve quality of life. Copper histadine is the only approved treatment that provides copper supplementation. However, it has limited effectiveness and new treatment options are being actively researched.

The Global Menkes Disease Market is estimated to be valued at USD 165.1 million in 2024 and is expected to reach USD 259.2 million by 2031, growing at a compound annual growth rate (CAGR) of 6.65% from 2024 to 2031.


Key Takeaways
Vtesse SNC, America Peptide Company, American Peptide Company are actively researching new treatment options. Vtesse SNC is conducting phase 3 trials of its gene therapy candidate VTX-009 while PeptaGen Pharmaceutical is developing copper nanoparticles.

Increased awareness about rare diseases and government support for orphan drug development is fueling demand for improved treatment options.

Advancements in the Menkes Disease Market gene therapy and precision medicine hold potential for developing curative therapies. Ongoing research on exon skipping and genome editing aims to correct the underlying genetic defect.

Market Trends
Gene therapy - Several gene therapy candidates are under development to deliver the corrected ATP7A gene and restore copper transport function. This represents a potentially curative approach.

Partnerships and collaborations - Companies are partnering with research institutes and patient advocacy groups to advance the drug development process and boost funding for rare disease research.

Market Opportunities
Orphan drug designation - Regulatory agencies offer incentives such as reduced fees, tax credits, and additional market exclusivity to support orphan drug development.

Patient advocacy - Increased awareness and support from patient advocacy groups encourages investment in developing effective treatments.

Impact of COVID-19 on Menkes Disease Market Growth
The COVID-19 pandemic has significantly impacted the Menkes Disease market. As per studies conducted during the pandemic, the market has witnessed a decline in growth owing toseveral factors. Strict lockdowns enforced by various governments led to disruptions in production and supply chains. This caused a delay in screening, diagnosis and treatment of Menkes disease patients. Restrictions on travel and transport further hampered supply of essential drugs and ingredients required for treatment.

With lockdowns being lifted gradually, the market is slowly recovering. However, it will take some time to reach pre-pandemic levels. Telemedicine emerged as a boon during this period and helped in continuity of care. Several virtual support groups were formed online to provide emotional support. Drug makers are catching up with production backlogs while ensuring appropriate safety protocols. Government initiatives to expedite screening programs will boost early detection rates. Collaborations between stakeholders can help address challenges in supply chain and ensure uninterrupted availability of treatment options.

Menkes Disease Market In Terms Of Value
Geographically, North America accounts for the largest share of the Menkes disease market in terms of value. This can be attributed to high awareness, developed healthcare infrastructure and presence of key market players. The United States dominates the regional market due to supportive reimbursement policies and focus on rare disease treatment. Asia Pacific is emerging as the fastest growing region for the Menkes disease market owing to rising income levels, growing medical tourism and increasing focus of global players.

Europe holds the second largest market share globally led by countries such as Germany, United Kingdom and France. Large patient pool, universal healthcare and focus on orphan drugs drive market growth. Meanwhile, Latin America and Middle East & Africa present lucrative opportunities for market expansion over the coming years. This is attributed to improving public healthcare spending, diagnosis rates and treatment seeking pattern in these regions.

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