Rockville, MD, March 18, 2025 (GLOBE NEWSWIRE) — According to Fact.MR, a market research and competitive intelligence provider, the The global rare neurological disease treatment market was valued at USD 41.8 billion in 2024 and has been forecasted to expand at a noteworthy CAGR of 9.7% to end up at USD 115.8 billion by 2035.
Technology-driven market expansion occurs in the global treatment Market because of government regulatory support and increased investment in gene and RNA-based therapeutic approaches.
Rare neurological disorder treatment markets exhibit transformative growth because researchers are approving gene therapies while spending more money on research and development. A Fact.MR analyst projects that CRISPR-based gene editing and antisense oligonucleotides (ASOs) and monoclonal antibodies will dominate the market within the next few years.
The rare neurological disease treatment sector expanded rapidly because of enrollment increases growing healthcare expenses and beneficial regulatory guidelines for developing novel treatments. Fast-tracking approvals and offering financial incentives to pharmaceutical companies have proven essential through the U.S. Orphan Drug Act and parallel policies adopted by the European Medicines Agency. Zolgensma (SMA treatment) along with Elevidys (DMD treatment) opened the path for gene therapy systems that scientists can use to treat neurological disorders. The pharmaceutical industry now offers broader treatment choices to patients through RNA-based drugs which include Spinraza for SMA and Qalsody for ALS.
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Food and Drug Administration has invested in artificial intelligence and machine learning systems that help researchers discover new drugs at an unprecedented speed.
Leading pharmaceutical corporations Biogen, Roche, and Novartis have significantly expanded their support for biotech start-ups to develop strategic partnerships concerning neurological research.
Early diagnosis of rare neurological diseases becomes more effective thanks to improved genetic testing as well as advanced screening methods which enhance patient awareness.
Continuous developments in biotechnology and artificial intelligence cause rapid growth in the rare neurological disease treatment market. A Fact.MR analyst predicts that
Key Takeaways from Market Study
- The global rare neurological disease treatment market is projected to grow at 7% CAGR and reach US$ 115.8 billion by 2035
- The market created an opportunity of US$ 12.1 billion growing at a CAGR of 9% between 2019 to 2024
- North America is a prominent region that is estimated to hold a market share of 8% in 2035
- Gene therapy under treatment type is estimated to grow at a CAGR of 4% creating an absolute $ opportunity of US$ 21.5 billion between 2025 and 2035
- North America and East Asia are expected to create an absolute $ opportunity of US$ 44.3 billion collectively
“Personalized Medicine Developments by Companies Will Create Promising Prospects for Rare Disease Treatment Progress.” says a Fact.MR analyst.
Leading Players Driving Innovation in the Rare Neurological Disease Treatment Market:
Pfizer, Inc; Novartis Pharmaceuticals Corp.; Merck & Co.; Johnson & Johnson Services, Inc.; Bayer AG; GlaxoSmithKline , Inc.; Sanofi S.A.; AbbVie Inc. (Allergan, Inc.); Teva Pharmaceuticals; F. Hoffmann-La Roche Ltd.; Other Prominent Players
Market Development
The market has expanded in treating rare neurological disorders yet access limitations and high prices create substantial barriers. Insurance companies together with government institutions are developing new payment models that enable access to costly healthcare treatments. The United States experience saw Medicare and private insurers establish outcome-based pricing systems that produce low-cost treatment opportunities for their beneficiaries.
Global governments enhance the pharmaceutical industry through orphan drug regulations as well as extended market protection and tax advantages for developing rare neurological condition treatments.
A $5 billion investment fund launched in 2023 provides financial support to Chinese local biotech firms developing cost-effective gene therapies for rare diseases. The reimbursement program supports the Chinese “Healthy China 2030” strategy by accelerating rare disease research along with treatment availability development.
Latest Market News & Developments
- FDA Approves New RNA Therapy for ALS (February 2024)
- Novartis Invests $3 Billion in Rare Neurological Disease Research (December 2023)
- China Approves First Domestically Developed Gene Therapy for SMA (October 2023)
Rare Neurological Disease Treatment Industry News:
- In July 2023, Biogen & Ionis collaborated on Next-Generation Gene Therapy for Huntington’s Disease using RNA interference (RNAi) technology to retard disease progression.
- In May 2023, The European Medicines Agency (EMA) sanctioned the first CRISPR therapy for a rare neurodegenerative disease, representing a breakthrough in treatments based on genome editing. This should give impetus to future advances in gene editing drugs.
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More Valuable Insights on Offer
Fact.MR, in its new offering, presents an unbiased analysis of the global rare neurological disease treatment market, presenting historical data for 2020 to 2024 and forecast statistics for 2025 to 2035.
The study reveals essential insights on the basis of the Indication (Neurodegenerative Disorders {Huntington’s Disease, Amyotrophic Lateral Sclerosis (ALS), Multiple System Atrophy, Lewy Body Dementia}, Neurodevelopmental Disorders {Rett Syndrome, Angelman Syndrome, Fragile X Syndrome}, Neuromuscular Disorders {Spinal Muscular Atrophy (SMA), Duchenne Muscular Dystrophy (DMD), Myasthenia Gravis}, Genetic and Metabolic Disorders {Wilson’s Disease, Niemann-Pick Disease, Tay-Sachs Disease}, Autoimmune Neurological Disorders {Guillain-Barré Syndrome (GBS), Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), Stiff-Person Syndrome}), Treatment Type (Gene Therapy, Cell Therapy, Enzyme Replacement Therapy (ERT), Small Molecule Drugs, Monoclonal Antibodies, Others), Drug Class (Biologics {Monoclonal Antibodies, Recombinant Proteins}, Small Molecule Drugs {Enzyme Inhibitors, Neuroprotective Agents}, RNA-Based Therapies {Antisense Oligonucleotides (ASO), siRNA-based Therapies}) Route of Administration (Oral, Injectable, Other Routes) Distribution Channel ( Hospital Pharmacies, Retail Pharmacies, Specialty Pharmacies, Online Pharmacies) across major regions of the world (North America, Latin America, Western Europe, Eastern Europe, East Asia, South Asia, and Pacific, Middle East & Africa).
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The global rare neurological disease treatment was valued at USD 41.8 billion in 2024 and has been forecasted to expand at a noteworthy CAGR of 9.7% to end up at USD 115.8 billion by 2035.
Currently, the global rare neurodegenerative disease treatment market accounts for a valuation of US$ 72.9 billion and is forecasted to reach US$ 119.2 billion by the end of 2033, expanding steadily at 5% CAGR over the next ten years.
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