Muckle-Wells Syndrome (MWS) refers to a rare inherited autoinflammatory disease, of the cryopyrin associated periodic syndromes (CAPS), caused by mutations in the NLRP3/ CIAS1 gene. The gene is associated with the coding of the protein cryopyrin. Mutations in the gene are said to result in the increased activity of a protein known as interleukin (IL) 1ß, which leads to inflammatory symptoms such as joint pain and fever. The syndrome is characterized by recurrent episodes of rashes, acute attacks of fever, conjunctivitis, and painful joint. In most cases, the syndrome also leads to progressive sensorineural hearing loss. Renal failure are also often associated in untreated conditions owing to the abnormal accumulation of the protein amyloid in the kidneys.
This report examines the market concerned with the diagnosis and treatment of MWS and presents a thorough overview of the present state of diagnostic tools and techniques and various drug compounds presently marketed for MWS. The report also includes detailed data regarding the state of ongoing research and development in the field, drugs in clinical trials and pipeline, and scope of growth for companies wanting to foray into the market.
Muckle-Wells Syndrome Market: Available Diagnostic and Therapeutics Options
As such, not many drug candidates are available to treat the condition completely. The few treatment options available in the market include Arcalyst (Rilonacept), an interleukin-1 blocker, by Regeneron Pharmaceuticals, Ilaris (Canakinumab), a monoclonal antibody to interleukin-1 beta, by Novartis Pharmaceuticals, and Kineret (Anakinra), an IL-1 receptor antagonist, by Biovitrum pharmaceuticals. Many drug candidates are also in pipelines.
For correcting the hearing loss that most MWS patients develop, hearing aids are prescribed. For alleviating joint pain, non-steroidal anti-inflammatory drugs are prescribed. The use of corticosteroids in high doses has also demonstrated positive effect in certain cases, but this may lead to several long- and short-term side effects.
For diagnosis of MWS, a thorough evaluation of the patient’s syndromes is done. For confirmation of the diagnosis, identification of NLRP3/ CIAS1 gene mutation is done. Identification of mutation is, however, not a sure shot way of confirming the diagnosis of MWS as not all patient possess this mutation.
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Muckle-Wells Syndrome Market: Drivers and Restraints
The market is expected to grow over the forecast period owing to advances in genetic engineering and increased focus of international pharmaceutical companies on research and development in the field of rare diseases such as MWS. Since MWS is a newly discovered condition, data regarding the actual prevalence and incidence are somewhat difficult to determine.
Also, there is a vast lack of awareness regarding the disease and R&D costs are high. These factors are expected to arrest the growth of the global MWS market to a large extent in the next few years. Nevertheless, government intervention, funds from private and public bodies, and attempts to raise awareness among the masses about the diseases could help the market gain traction.
Muckle-Wells Syndrome Market: Competitive Landscape
The vendor landscape of the global MWS market features few players and a low level of competition, typical of most new markets. More companies are expected to enter the market via experimental drugs in the next few years. Better diagnostic methods are expected to be devised owing to increased vendor base in the market and advances in genetic engineering. Some of the key vendors presently operating in the market are Novartis AG, Charite University, Berlin, Germany, Istituto Giannina Gaslini, and Regeneron Pharmaceuticals, Inc.
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