The global Leigh syndrome treatment market is estimated to grow rapidly over the forecast period of 2024 to 2031. Leigh syndrome, also known as subacute necrotizing encephalomyelopathy, is a rare genetic neurological disorder that affects the central nervous system and predominantly seen in infants and children under 2 years of age. Treatment of Leigh syndrome primarily focuses on improving neurological deficits and managing associated symptoms. Therapy includes dietary patterns, drugs, and supportive treatments, depending on the specific deficiencies in the patient.
The global Leigh syndrome treatment market is driven by the growing demand for novel therapeutics and targeted treatment options.
The Global Leigh Syndrome Treatment Market is estimated to be valued at US$ 272.9 Mn in 2024 and is expected to exhibit a CAGR of 6.7% over the forecast period 2024 to 2031.
Key Takeaways
Key players: Abliva AB and PTC Therapeutics are among the leading players developing gene therapies for Leigh syndrome treatment.
Growing demand: The increasing prevalence of rare genetic disorders like Leigh syndrome and rising awareness about management options are fueling the Leigh Syndrome Treatment Market share and the demand for effective treatment therapies.
Global expansion: Key participants are expanding their regional presence through collaboration with regional healthcare providers to improve access to innovative Leigh syndrome therapies globally.
Market key trends
Novel drug development is a major trend in the global Leigh syndrome treatment market. Companies are conducting clinical trials to develop gene therapies and targeted drug formulations with improved efficacy and safety profile. For instance, Abliva AB is evaluating its investigational gene therapy AB-101 in a phase I/II clinical trial for patients with Leigh syndrome. In addition, strategic collaborations between pharma companies and research institutes are speeding up drug discovery efforts to address unmet needs in Leigh syndrome therapeutics.
Porter’s Analysis
Threat of new entrants: New entrants face high costs associated with R&D investment required to develop new effective treatments for this rare genetic disorder. Stringent regulations from regulatory authorities also pose significant entry barriers.
Bargaining power of buyers: The bargaining power of buyers is moderate as treatment options are limited for this rare disease however healthcare providers have influence over treatment decisions.
Bargaining power of suppliers: Suppliers having novel treatment offerings or late stage pipeline candidates hold strong bargaining power due to limited treatment alternatives currently available.
Threat of new substitutes: Threat of new substitutes is high as research continues to explore new treatment modalities for Leigh syndrome such as gene therapies, neuroprotective therapies, and other targeted approaches.
Competitive rivalry: Leigh Syndrome Treatment Market Challenges and Opportunities include moderate to high competition as existing players strive to develop more effective therapies and extend their patent exclusivity duration through ongoing clinical research.
Geographical regions
North America currently dominates the global Leigh syndrome treatment market in terms of value share. This is attributed to rising disease awareness, growing research activity, and availability of advanced healthcare facilities in the region.
Fastest growing region:
Asia Pacific region is poised to witness highest growth during the forecast period driven by increasing patient population, expanding healthcare spending, and growing efforts toward rare disease treatment in regional hubs like China, India, and Japan.
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