A market study presented by FMI ‘Mucopolysaccharidosis Treatment Market: Global Industry Analysis 2014-2018 and Opportunity Assessment 2019-2029,’ explains the significant factors influencing the current market structure.
According to research, the mucopolysaccharidosis treatment is still emerging, and different geographies have implemented standard treatment options for the condition. However, there is no universally accepted treatment pattern for mucopolysaccharidosis. Mucopolysaccharidosis falls under rare diseases, which is a complex, diverse, constantly evolving field, and there is a significant shortage of medical and scientific data related to it. Mucopolysaccharidosis treatment and diagnosis involves complex managing requirements, which include long-term care, rehabilitation support, and a continuous treatment plan.
Research Activities Uplift Stem Cell Therapy Application in MPS Treatment
In the present scenario, the drugs that are considered for mucopolysaccharidosis treatment, such as aldurazyme, naglazyme, vimizim, elaprase, mepsevii and hunterase, are the only regulated and recommended drugs present in the market. These drugs fall under enzyme replacement therapies, however, with present research initiatives for stem cell therapies, the latter is considered a prominent mucopolysaccharidosis treatment. Clinical research shows that stem cell transplantation covers a large area that is not covered with the more frequently recommended practice of enzyme replacement therapies, which changes the current market structure for mucopolysaccharidosis treatment, prioritising the latter.
Stem cell therapy is a therapeutic option for mucopolysaccharidosis patients suffering from a severe phenotype, as research shows the method can preserve neurocognition or can even help break the progressive neurodegeneration. The method is provided with strict selection criteria, which is followed by maintained regulations. Research shows that stem cell therapy as a treatment option is gaining popularity among healthcare professionals for mucopolysaccharidosis treatment, which can be attributed to the relation of its better reach towards a normal health condition for the patient
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Enzyme Replacement Therapy Remains Lucrative for Market Investors
As per further assessments of the mucopolysaccharidosis treatment market, it has been difficult to collect epidemiological data pertaining to rare diseases, especially mucopolysaccharidosis treatment, which is likely to impede the actual estimation of the economic burden associated with the condition. The cost estimation for each mucopolysaccharidosis treatment type is affecting and, in turn, creating a more vulnerable situation for the businesses and disrupting research & development activities for each company.
Enzyme replacement therapy is the most attractive segment for investors, however, the unavailability of reimbursement plans and precise treatment plans is encouraging most patient in developing regions to opt for symptomatic treatments instead of enzyme replacement therapy, which is still considered to be the standard mucopolysaccharidosis treatment.
Key Players Focus on Clinical Research of Treatment Models
The report segments the mucopolysaccharidosis treatment market into seven regions to elaborate on the regional trends pertaining to the treatment plan. Major players are focusing on increasing their market share in the Asia Pacific market through strategic collaborations with regional research institutes. There is a lack of awareness about rare diseases among the general public as well as in medical healthcare facilities in several emerging economies. According to a survey and industry report, it takes patients in the US an average of 7.6 years and patients in the UK an average of 5.6 years to actually receive a proper diagnosis. Moreover, it involves a team of healthcare professionals to actually get the right mucopolysaccharidosis treatment and diagnosis pattern for reported cases.
Manufacturers are in the process of introducing a considerable number of mucopolysaccharidosis treatment options, which are currently under clinical trials. An estimated 160 and above clinical trials are being performed for mucopolysaccharidosis treatment. Thus, ensuring that the manufacturers take this mucopolysaccharidosis treatment market to be lucrative and potential rich in terms of revenue. Medical research institutes play an important role in this particular market. They are expected to be the bridge between treatment plans and economical solutions for manufacturers, thus leaving an explicable and lucrative model for mucopolysaccharidosis treatment.
The mucopolysaccharidosis treatment market includes companies such as BioMarin Takeda Pharmaceutical Company Limited, Sanofi S.A., and Ultragenyx Pharmaceutical Inc. BioMarin accounts for a significant value share in the present mucopolysaccharidosis treatment market. The currently existing promising drug types are expected to face competition from emerging candidates. In addition the mucopolysaccharidosis treatment products of other companies, such as Sangamo Therapeutics, Inc., REGENXBIO Inc., Sarepta Therapeutics, Abeona Therapeutics, Inc., and others, are also in the pipeline.
By Treatment Type
- Enzyme Replacement Therapies
- Stem Cell Therapies
- Bone Marrow Transplantation
- Umbilical Cord Blood Transplantation
By Type of MPS
- MPS I
- MPS II
- MPS IV A
- MPS VI
- MPS VII
By End User
- Specialty Clinics
- Medical Research Centers
- North America
- Latin America
- South Asia
- East Asia
- Middle East & Africa
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