Number of pages: 100 | Report Format: PDF | Published date: April 24, 2023
Historical Years – 2021 | Base Year – 2022 | Forecasted Years – 2023-2031
Report Attribute |
Details |
CAGR |
4.95% |
Base Year for Estimation |
2022 |
Forecast Period |
2023 to 2031 |
Historical Year |
2021 |
Segments Covered |
Type, Treatment Type, and Region |
Regional Scope |
North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa |
According to the deep-dive market assessment study by Growth Plus Reports, the global limb-girdle muscular dystrophy treatment market is expected to register a revenue CAGR of 4.95% from 2023 to 2031.
Limb-Girdle Muscular Dystrophy Treatment Market Fundamentals
Limb-girdle muscular dystrophy (LGMD) is an illness with various subgroups classified by disease gene and inheritance. LGMD often appears in the proximal muscles around the hips and shoulders. (The proximal muscles are those closest to the body’; distal muscles are farther distant from the center — for instance, in the hands and feet). The shoulder girdle is the bony structure that surrounds the shoulder area, while the pelvic girdle is the bony structure that surrounds the hips. The limb girdles are a set of illnesses named after the apparent weakening and atrophy (wasting) of the muscles associated with the limb girdles. LGMDs are distinguished by the weakening and atrophy of the limb-girdle muscles. Additional symptoms distinguish some LGMD subtypes. Some kinds of LGMD, for instance, might cause heart muscle weakening (cardiomyopathy) and/or incorrect transmission of signals that regulate the heartbeat (conduction abnormalities or arrhythmias). Some disease subtypes also affect the muscles used for breathing. Therefore, respiratory function, along with heart function, should be checked regularly. Other symptoms of LGMD might involve but are not limited to joint stiffness, muscular cramping, calf muscle hypertrophy, and involvement of distal muscles of the body, such as those controlling the hands and feet.
The limb-girdle muscular dystrophy is further sub-classifies into:
The treatment for limb-girdle muscular dystrophy includes pharmacological therapy to relieve symptoms (corticosteroids, gene therapy, stem cell therapy), physical therapy for muscle weakness, respiratory therapy for breathing issues, and occupational therapy for swallowing difficulties and other medical care.
Limb-Girdle Muscular Dystrophy Treatment Market Dynamics
The growth of the limb-girdle muscular dystrophy treatment market is expected to show considerable revenue growth from 2023 to 2031, majorly attributed to the growing research and development initiatives for developing the treatment for such a rare disease. For instance, they are growing pipeline drugs such as BBP-418 (ribitol), SRP-9003, GenPHSat, and LION-101. Additionally, the researchers are exploring gene therapy to treat LGMD by introducing functional copies of the affected genes into patients’ cells. Several gene therapy products are currently in development, with some showing promising results in early-stage clinical trials. Some drugs are being investigated as potential treatments for LGMD. Researchers are also exploring the use of stem cells to treat LGMD. For instance, a clinical trial is underway to test the safety and efficacy of using mesenchymal stem cells to treat LGMD2I. In addition to these specific treatments, supportive care is important in managing LGMD. This also includes physical therapy, occupational therapy, and other interventions aimed at improving the quality of life for patients. Several initiatives are underway to encourage collaboration between researchers, clinicians, patients, and other stakeholders in the LGMD community. Pharmaceutical companies are investing heavily in research and development to develop new treatment options for LGMD. This is expected to drive the growth of the LGMD treatment market in the coming years. Governments and non-governmental organizations are working to raise awareness about LGMD and the need for new treatment options. This is expected to drive the demand for LGMD treatments in the future. Regulatory bodies, such as the US FDA and the European Medicines Agency, support companies developing new LGMD therapies. This is expected to accelerate the approval process for new treatments and drive the growth of the LGMD treatment market.
However, several challenges can hamper the revenue growth of the limb-girdle muscular dystrophy treatment market. There are currently no FDA-approved treatments for LGMD, which makes it challenging for patients to manage their symptoms effectively. LGMD is a rare disease, and it can take a long time for patients to receive an accurate diagnosis. This delay can result in a delay in starting appropriate treatment. Developing a treatment for a rare disease is expensive, which makes it challenging for pharmaceutical companies to invest in research and development. LGMD affects a relatively small number of people, which makes it difficult to conduct large-scale clinical trials or attract investment from pharmaceutical companies. LGMD is a diverse group of disorders with many subtypes, each with unique genetic mutations and clinical manifestations. This heterogeneity makes it challenging to develop a universal treatment that can be effective for all patients. Patients with LGMD may have limited access to specialized care, including genetic counseling and physical therapy, which can impact their quality of life and overall prognosis.
Limb-Girdle Muscular Dystrophy Treatment Market Ecosystem
The global limb-girdle muscular dystrophy treatment market is analyzed from three perspectives: type, treatment type, and region.
Limb-Girdle Muscular Dystrophy Treatment Market by Type
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Based on the type, the global limb-girdle muscular dystrophy treatment market is segmented into limb-girdle muscular dystrophy type A (Dominant) and limb-girdle muscular dystrophy type B (Recessive).
The limb-girdle muscular dystrophy type A (Dominant) is estimated to account for a significant revenue share in the global market. LGMD A is one of the most common subtypes of LGMD, and its prevalence is increasing globally. This rising prevalence is expected to drive demand for LGMD A treatments in the coming years. Advances in genetic testing have enabled earlier and more accurate diagnoses of LGMD A, which can help identify patients who may benefit from treatment. There is a growing interest among pharmaceutical companies in developing treatments for LGMD A, which has resulted in increased investment in research and development. Regulatory agencies such as the FDA and the EMA are also showing great interest in providing expedited review and approval for treatments for rare diseases like LGMD A, which can encourage companies to invest in this market. While there are currently no FDA-approved treatments for LGMD A, several promising therapies in clinical development have shown potential in early-stage trials. These advances in treatment options are expected to drive growth in the LGMD A treatment market in the coming years.
Limb-girdle muscular dystrophy (LGMD) type B (Recessive) segment accounts for considerable revenue share and is expected to show slow growth during the forecast period. Limb-girdle muscular dystrophy (LGMD) type B (Recessive) is a rare form of the disease caused by a deficiency of a protein called dysferlin. There are currently no FDA-approved treatments for LGMD type B, highlighting the urgent need for effective treatment options. This creates an opportunity for companies to invest in developing novel therapies. Advances in understanding the pathophysiology of LGMD type B have led to the development of targeted treatment approaches that address the underlying genetic defect. The prevalence of LGMD type B is increasing, which is expected to create a larger patient population needing treatment during the forecast period.
Limb-Girdle Muscular Dystrophy Treatment Market by Treatment Type
Based on the patient type, the global limb-girdle muscular dystrophy treatment market is segmented into corticosteroids, gene therapy, stem cell therapy, and others.
The corticosteroids segment accounts for a significant revenue share in the global market. Corticosteroids are a medication that can reduce inflammation and slow muscle degeneration in patients with limb-girdle muscular dystrophy (LGMD). Several factors drive the growth of the corticosteroids segment in the global LGMD treatment market. Corticosteroids effectively slow the progression of LGMD in some patients, leading to their widespread use as a standard of care in many countries. Prednisone and deflazacort (Emflaza) are corticosteroids commonly used to treat limb-girdle muscular dystrophy (LGMD). These medications work by reducing inflammation and slowing the progression of muscle degeneration in patients with LGMD. They have improved some patients’ muscle strength, function, and quality of life. The prevalence of LGMD is increasing globally, driving demand for effective treatment options, including corticosteroids. Corticosteroids are relatively inexpensive compared to other treatment options, which can make them an attractive option for patients and healthcare providers.
Similarly, the gene therapy segment is also expected to notably during the projected period from 2023 to 2031. Genetic advances have led to a better understanding of the genetic mutations responsible for LGMD, enabling the development of targeted gene therapies. Regulatory agencies such as the FDA have shown a willingness to provide expedited review and approval for gene therapies for rare diseases like LGMD, which can encourage companies to invest in this market. Advances in gene delivery technologies have made it easier and safer to deliver therapeutic genes to target cells, which has increased the feasibility of gene therapy for LGMD. Several gene therapy clinical trials for LGMD have shown promising results, including improvements in muscle function and reduced muscle degeneration.
Limb-Girdle Muscular Dystrophy Treatment Market by Region
Based on the region, the global limb-girdle muscular dystrophy treatment market is segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa.
The region’s well-developed healthcare infrastructure includes advanced diagnostic tools and treatment options, which can facilitate early diagnosis and optimal management of LGMD. The US FDA has shown a willingness to provide expedited review and approval for treatments for rare diseases like LGMD, which can encourage companies to invest in this market. North America has high healthcare spending per capita, which can facilitate access to expensive treatments like gene therapy. The region is home to many leading research institutions and biotechnology companies actively developing new treatment approaches for LGMD.
Competitive Landscape of Limb-Girdle Muscular Dystrophy Treatment Market
The prominent players operating in the global limb-girdle muscular dystrophy treatment market are:
Strategic Development in Limb-Girdle Muscular Dystrophy Treatment Market
The corticosteroid segment dominates the global limb-girdle muscular dystrophy treatment market with the largest revenue share.
The global limb-girdle muscular dystrophy treatment market is expected to register a revenue CAGR of 4.95% during the forecast period.
Asia Pacific is expected to be the fastest-growing limb-girdle muscular dystrophy treatment market revenue share.
Jubilant Cadista Pharmaceuticals Inc., Aden Pharmaceuticals Private Limited, ML Bio Solutions Inc., and Genethon are key market players operating in the limb-girdle muscular dystrophy market.
Limb-girdle muscular dystrophy is a group of illnesses that cause muscle weakening and atrophy in the arms and legs. The muscles nearest to the body (proximal muscles) are the most impacted, notably the muscles of the shoulders, upper arms, pelvic region, and thighs.
*Insights on financial performance are subject to the availability of information in the public domain