Segments - Limb-girdle Muscular Dystrophy Treatment Market by Dystrophy Type (Limb-girdle Muscular Dystrophy Type-A and Limb-girdle Muscular Dystrophy Type-B), Treatment Type (Symptomatic Treatments, Molecular & Gene Therapy, Stem-Cell Transplantation, Exon Skipping, and Others), End-user (Hospital, Ambulatory Centers, and Others), and Region (Asia Pacific, North America, Latin America, Europe, and Middle East & Africa) - Global Industry Analysis, Growth, Share, Size, Trends, and Forecast 2024 – 2032
The global limb-girdle muscular dystrophy treatment market size was USD XX Million in 2023 and is likely to reach USD XX Million by 2032, expanding at a CAGR of XX% during 2024–2032. The market growth is attributed to the increasing risk of limb-girdle muscular dystrophy and rising investment in pharmaceutical manufacturing.
A broad range of illnesses with numerous subgroups classified by heredity and disease gene make up limb-girdle muscular dystrophy (LGMD). Primitive muscles in the vicinity of the hips and shoulders are typically affected by LGMD. Proximal muscles are located closest to the center of the body, while distal muscles are located farther away and are found, for instance, in the hands and feet.
The bony structure encircling the shoulder is called the shoulder girdle, while the bony structure encircling the hips is called the pelvic girdle. These are collectively referred to as the limb-girdles, and the term limb-girdles dystrophy refers to the observable weakening and atrophy (wasting) of the muscles associated with the limb-girdles. When limb-girdle muscular dystrophy begins in childhood, the progression appears to be fast and the disease increasingly disabling.
According to a report, the collective illnesses that make up LGMD represent the fourth most prevalent hereditary cause of muscle weakness, with an approximate prevalence of 2 per 100,000 people.
The treatment of limb-girdle dystrophy focuses on maintaining function and preventing contractures. The American Academy of Neurology recommends cardiac evaluation for patients at high risk of cardiac complications and pulmonary function testing for those at high risk of respiratory failure. All the concerned patients are referred to a multispecialty clinic with neuromuscular disorder expertise.
Limb-girdle muscular dystrophy is inherited through recessive or dominant inheritance. Recessive inheritance occurs when a person receives the genetic mutation from both biological parents, while dominant inheritance requires only one gene from one parent. This type is found in myotonic, facioscapulohumeral, and oculopharyngeal muscular dystrophies. Sex-linked inheritance occurs when a man has one X and one Y chromosome, and a female has two X chromosomes. A genetic mutation on the X chromosome causes a disease, while X-linked illnesses occurs in genetically feminine individuals, with milder symptoms. This type of inheritance is common in Duchenne and Becker's muscular dystrophies.
Limb-girdle muscular dystrophy (LGMD) is a heterogeneous group of muscle disorders with no current cure. However, Artificial Intelligence (AI) has the potential to revolutionize the LGMD treatment market by significantly impacting the drug development and design process, offering a glimmer of hope for future therapeutic breakthroughs. AI analyzes the genetic, molecular, and clinical data to identify novel drug targets specific to different LGMD subtypes. This expedites the discovery of potential therapeutic avenues that were previously overlooked using traditional methods.
AI is able to perform in-silico simulations to virtually screen millions of candidate molecules and identify those with the most promising therapeutic potential for LGMD. This significantly reduces the time and resources needed for traditional drug discovery processes. AI is paving the way for personalized treatment plans that target the underlying cause of their LGMD subtype by analyzing individual patient genetic data to predict their response to specific therapies.
On July 12, 2023, Thales, a Europe-based developer of artificial intelligence for mission-critical systems, and Genethon, an innovator & key company in genetic therapy development and research for rare genetic diseases, are combining the expertise to create the first-of-its-kind effective digital models for enhancing bioproduction produces in the field of gene therapy. The process of developing a gene therapy medication is incredibly complex, costly, time-consuming, and tough. One of the major challenges is cutting production time and expenses.
Increasing risk of limb-girdle muscular dystrophy is driving the market. The primitive muscles in the vicinity of the hips and shoulders are typically affected by LGMD and cause a lot of health issues. Furthermore, growing awareness among population and increasing government initiatives for the disease awareness are rising the demand for a preventive treatment, boosting the market.
Increasing R&D activities for drug development is expected to drive the global market during the forecast period. Increasing interests of researchers and scientists to find out the cure before the competitors fuel the market. Moreover, several pipeline drugs such as SRP-9003, GenPHSat, BBP-418 (ribitol), and LION-101 are expected to receive the FDA approval and to be launched in the market in the coming years, boosting the market.
Unavailability of any FDA approved drug is expected to restrain the market during the forecast period. While the pandemic did not significantly impact drug approvals in 2020 and 2021,the slow implementation processes of the pharmaceutical industry contribute to the stability of approval numbers over time. Despite challenges, the industry continues to innovate, ensuring that patients have access to effective medications.
Development of treatment medication is expected to drive the global market and open up the opportunities for manufacturers and patients during the forecast period. Increasing establishment of manufacturing companies and major market players launching FDA approved drugs are anticipated to propel the global market during the predicted period. Growing demand, technological advancements, and government policies drive the manufacturing companies producing FDA-approved drugs. Increasing number of companies are incentivized to enter the market, as the demand for these drugs increases. Governments are offering incentives or subsidies to encourage domestic manufacturing of FDA-approved drugs.
Major market players, such as established pharmaceutical companies, are launching new drugs after FDA approval, contribute to the market growth by having strong brand recognition, extensive research & development (R&D) investments, and increased investor confidence. These factors help to quickly deliver new FDA-approved drugs to patients and attract investments to the market, fueling further development and innovation.
The market report includes an assessment of the market, trends, segments, and regional markets. Overview and dynamics have also been included in the report.
Attributes |
Details |
Report Title |
Limb-girdle Muscular Dystrophy Treatment Market - Global Industry Analysis, Growth, Share, Size, Trends, and Forecast |
Base Year |
2023 |
Historic Data |
2017–2022 |
Forecast Period |
2024–2032 |
Segmentation |
Dystrophy Type (Limb-girdle Muscular Dystrophy Type-A and Limb-girdle Muscular Dystrophy Type-B), Treatment Type (Symptomatic Treatments, Molecular & Gene Therapy, Stem-Cell Transplantation, Exon Skipping, and Others), and End-user (Hospital, Ambulatory Centers, and Others) |
Regional Scope |
Asia Pacific, North America, Latin America, Europe, and Middle East & Africa |
Report Coverage |
Company Share, Market Analysis and Size, Competitive Landscape, Growth Factors, Market Trends, and Revenue Forecast |
Key Players Covered in the Report |
Asklepios BioPharmaceutical Inc.; Atamyo Therapeutics; BridgeBio Inc. and Hansa Biopharma.; Edgewise Therapeutics; Genethon; Jubilant Cadista Pharmaceuticals Inc.; ML Bio Solutions; Sarepta Therapeutics Inc.; Simone Spuler MD; Vita Therapeutics Inc. |
Based on dystrophy type, the limb-girdle muscular dystrophy treatment market is divided into limb-girdle muscular dystrophy type-A and limb-girdle muscular dystrophy type-B. The limb-girdle muscular dystrophy type-A segment accounts for 55% of the total market share and is expected to expand at a significant growth rate during the projection period. The segment is dominating the global market, as it is the most prevalent type of limb-girdle muscular dystrophy. It is estimated that about 30% of total cases of limb-girdle muscular dystrophy are Type 2A. The type-A is the dominant inheritance and in this, the person only needs to get inherited with the defective genes from parents.
On the basis of treatment type, the global market is segregated into symptomatic treatments, molecular & gene therapy, stem-cell transplantation, exon skipping, and others. The symptomatic treatments segment is anticipated to register a considerable CAGR in the coming years, owing to the fast reduction of inflammations and other symptoms. Symptomatic drugs slow downs the disease progression and slow the muscular degradation as well. Prednisone and deflazacort (Emflaza) are corticosteroids (symptomatic drug) commonly used to treat limb-girdle muscular dystrophy (LGMD). Additionally, symptomatic drugs are a comparatively cheaper option as compared to other treatment options, which boost the segment.
On the basis of end-users, the Limb-girdle muscular dystrophy treatment market is segmented into hospital, ambulatory centers, and others. The hospital segment is expected to register a robust growth rate during the forecast period, due to the number of facilities provided in hospitals.
Moreover, the comprehensive care facilities and a wide range of medical services that are available in the hospitals boost segment. Hospitals offer specialized care, access to advanced therapies, and multidisciplinary teams comprising neurologists, physiotherapists, & genetic counselors for LGMD patients. LGMD patients often require hospitalization for acute exacerbations, complications, or surgical interventions. Hospitals offer inpatient services, including surgeries, respiratory support, and rehabilitation, which contribute to the segment growth.
In terms of region, the global Limb-girdle muscular dystrophy treatment market is classified as Asia Pacific, North America, Latin America, Europe, and the Middle East & Africa. North America is expected to dominate the market with 40% of total market share during the forecast period, owing to the advanced medical infrastructure. Modern medical facilities and cutting-edge treatment options in the region contribute to its leadership in treating limb-girdle muscular dystrophy. Additionally, significant healthcare investments and high-income levels in North America drive the demand for effective treatments, including those for muscular dystrophy. Furthermore, renowned research institutes and companies in North America actively contribute to advancements in limb-girdle muscular dystrophy treatments. Collaborations, regulatory approvals, and technological advancements are expected to drive the market in the region during the forecast period.
The limb-girdle muscular dystrophy treatment market has been segmented on the basis of
Key players competing in the global Limb-girdle muscular dystrophy treatment market are Asklepios BioPharmaceutical Inc.; Atamyo Therapeutics; BridgeBio Inc. and Hansa Biopharma.; Edgewise Therapeutics; Genethon; Jubilant Cadista Pharmaceuticals Inc.; ML Bio Solutions; Sarepta Therapeutics Inc.; Simone Spuler MD; Vita Therapeutics Inc.
These companies adopted development strategies including mergers, acquisitions, partnerships, collaboration, product launches, and production expansion to expand their consumer base globally. For instance,
On August 3, 2023, Asklepios BioPharmaceutical, Inc., a genetic therapy company fully owned and independently operated as a subsidiary of Bayer AG, announced that the first participant in the Phase 1 / Phase 2 LION-CS101 research study for individuals with limb-girdle muscular dystrophy subtype 2I/R9 (LGMD2I/R9) was recently dosed. A novel FKRP replacement gene therapy known as AB-1003 (also known as LION-101) is currently being studied.
On June 22, 2023, According to Sarepta Therapeutics, Inc., the pioneer in precision genetic therapy for rare diseases, the FDA expedited the authorization of delandistrogene moxeparvovec-rokl (ELEVIDYS), an adeno-associated virus-based gene therapy. ELEVIDYS is intended for the management of ambulatory children and adolescents with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene. This indication received fast approval based on the levels of ELEVIDYS micro-dystrophin seen in patients treated with ELEVIDYS.