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DUBLIN, Nov. 25, 2022 /PRNewswire/ -- The "Repeat Expansion Disorders Treatment Market Size, Market Share, Application Analysis, Regional Outlook, Growth Trends, Key Players, Competitive Strategies and Forecasts, 2022 To 2030" report has been added to ResearchAndMarkets.com's offering.
The market for repeat expansion disorders treatment is expected to grow at a CAGR of 13.5% during the forecast period of 2022 to 2030.
Increasing Research of Rapid Expansion Disorders Therapies is Boosting the Market
Because of the complexity of repetitive disease genetics and pathobiology, previously unknown commonalities and molecular mechanisms associated with the diseases, such as RNA toxicity, have been uncovered. The investigation of polyglutamine diseases has also shown that post-translational modification is an important step in the pathogenic cascade, and it has demonstrated that the autophagy pathway plays an important role in the degradation of misfolded proteins. These are two themes that are likely to be applicable to the entire field of neurodegeneration.
Research on recurrent diseases is yielding insights that are sparking new research avenues. These new lines of inquiry should not only elucidate the molecular mechanisms of disease, but also highlight opportunities for therapeutic intervention in the treatment of conditions that are currently incurable. Despite of these advancements, there are challenges - just as there were when the science was just starting out - in pinpointing the precise manner in which repetitive expansion mutations lead to inherited disorders. As a result, the classification schemes that are currently in use run the risk of becoming obsolete in the near future.
Market to Present Significant Opportunities During the Forecast Period
Since the first discovery of a novel and obscure type of mutation as the root cause of fragile X syndrome and spinal muscular atrophy (SBMA), a significant amount of progress has been made. The research that has been done in this area has uncovered an extraordinary unanticipated complexity, which includes toxicities that are mediated at the protein, RNA, and genome levels. Research on recurrent diseases, for instance, continues to be one of the most important factors in determining how faulty RNA metabolism and autophagy contribute to human illness.
Why the development of relevant treatments has not kept pace with the extraordinary trajectory of scientific advance in the understanding of mechanisms and pathways is an issue that keeps coming up in the field of recurrent disease. This disconnect applies to the vast majority of neurological disorders; nevertheless, repeat diseases present a fantastic potential for the development of reasonable therapies due to the fact that conclusive diagnosis is typically predicated upon repeat allele expansion. These discoveries have also led to some very encouraging therapeutic developments, such as the utilisation of oligonucleotide and morpholino anti-sense repeats targeted against the DM1 CTG expansion.
Huntington Disease Treatment Market Presenting Largest Opportunities
Huntington disease treatment market among other rapid expansion disorders treatment is expected grow the fastest registering a CAGR of 20% during the forecast period 2022 to 2030. It is estimated that important drivers during the forecast period will include the planned label expansion of Ingrezza for the treatment of chorea associated with Huntington's disease, the high burden of HD in western countries, and a robust product pipeline of disease-modifying treatments. There are around 30,000 persons in North America who have HD, and the disease has a prevalence of 5.7 cases per 100,000 people. HD in children is significantly less common than in adults, accounting for only approximately 5 - 10% of all cases. It affects as many as 10 people out of every 1,00,000 people in Europe.
Market Segmentation
Disorders
- Dentatorubral-pallido-luysian atrophy (DRPLA)
- Fragile X syndrome ofmental retardation
- Fragile X tremor ataxia syndrome (FXTAS)
- FRAXE mental retardation (FMR2)
- Spinal and Bulbar Muscular Atrophy/Kennedy's Disease
- Huntington's Disease
- Oculopharyngeal musculardystrophy (OPMD)
- Myoclonic epilepsy
- Myotonic dystrophy
- Spinocerebellar ataxia
- Friedreich Ataxia
- Amyotrophic lateral sclerosis (ALS)
- Frontotemporal dementia (FTD).
- Myoclonic epilepsy
- Syndromic / non-syndromicX-linked mental retardation(Gene: ARX)
Route of Administration
Distribution Channel
- Hospital Pharmacies
- Retail Pharmacies
- Online Pharmacies
Drug Pipeline (Profile)
- Spinal and Bulbar Muscular Atrophy/Kennedy's Disease
- Huntington's Disease
- spinocerebellar ataxia
- Friedreich Ataxia
- amyotrophic lateral sclerosis (ALS)
- frontotemporal dementia (FTD)
Region Segment (2020 - 2030; US$ Million)
Key Topics Covered:
1. Preface
2. Executive Summary
3. Repeat Expansion Disorders Treatment Market: Business Outlook & Market Dynamics
4. Repeat Expansion Disorders Treatment Market: By Disorders, 2020-2030, USD (Million)
5. Repeat Expansion Disorders Treatment Market: By Route of Administration, 2020-2030, USD (Million)
6. Repeat Expansion Disorders Treatment Market: By Distribution Channel, 2020-2030, USD (Million)
7. Repeat Expansion Disorders Treatment Market: By Drug Pipeline (Profile), 2020-2030, USD (Million)
8. North America Repeat Expansion Disorders Treatment Market, 2020-2030, USD (Million)
9. UK and European Union Repeat Expansion Disorders Treatment Market, 2020-2030, USD (Million)
10. Asia Pacific Repeat Expansion Disorders Treatment Market, 2020-2030, USD (Million)
12. Middle East and Africa Repeat Expansion Disorders Treatment Market, 2020-2030, USD (Million)
13. Company Profile
Companies Mentioned
- Pfizer Inc.
- CRISPR Therapeutics
- Acorda Therapeutics
- Capsida Biotherapeutics Inc.
- Larimar Therapeutics Inc.
- Healx
- Intellia Therapeutics Inc.
- Editas Medicine
- Bluebird bio Inc.
- Sanofi
- Merck & Co. Inc.
- H. Lundbeck A/S
- Cellectis SA
- Bio-Techne
- GlaxoSmithKline plc
- Allergan Aurobindo Pharma Ltd.
- Sun Pharmaceutical Industries Ltd.
- Torrent Pharmaceuticals Ltd.
- Intas Pharmaceuticals Ltd.
- Cipla Ltd
For more information about this report visit https://www.researchandmarkets.com/r/ibmrey
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