Promising clinical research could revolutionise mucopolysaccharidosis treatment

Promising clinical research could revolutionise mucopolysaccharidosis treatment
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According to research, mucopolysaccharidosis treatment is still emerging, but recent drug approval & promising clinical research studies could accelerate treatment pattern for those suffering.

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. Treatment and diagnosis involve complex managing requirements, which include long-term care, rehabilitation support, and a continuous treatment plan. Different geographies have implemented standard options for mucopolysaccharidosis treatment, however, there is no universally accepted treatment pattern for mucopolysaccharidosis.

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.

Such 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.

Enzyme replacement therapy remains lucrative

As per further assessments of the MPS 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 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 treatment.

Key players focus on clinical research of treatment models

Manufacturers are in the process of introducing a considerable number of options for treatment, 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.

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.

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