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Global Andersen-Tawil Syndrome Market reached US$ 1.9 billion in 2022 and is expected to reach US$ 3.3 billion by 2030, growing with a CAGR of 7.4% during the forecast period 2023-2030.
Andersen-Tawil syndrome is an inherited disorder caused by a mutation in the KCNJ2 gene. This gene encodes an ion channel that transports potassium ions out of the cardiac muscle cells. This ion channel is essential for maintaining the normal functions of the skeletal and cardiac muscles. It is usually inherited from a parent in an autosomal dominant manner, but it can also occur due to a new genetic mutation. The specific symptoms and severity can vary greatly from one person to another, even among members of the same family.
Furthermore, the global Anderson-tawil syndrome market is driven by various factors like an increase in the prevalence of Andersen-tawil syndrome, increasing focus of the government to prioritize drugs for the treatment of rare diseases, increasing participation of organizations to support the treatment and to create awareness, rising research and development investments by the key players with having key players like Medtronic Plc, Abbott Laboratories, Boston Scientific Corp, LivaNova Plc and others.
The rise in the prevalence of Andersen Tawil Syndrome is one of the key factors that helps the market to grow during the forecast period. Andersen-Tawil syndrome (ATS) is a rare autosomal dominant disorder with recurrent flaccid muscle weakness, cardiac arrhythmias, and distinctive skeletal and facial features. The prevalence is estimated at 0.8-1 in 1,000,000, but the actual prevalence is expected to be higher due to the reported prevalence from a specific population and subgroup of ATS patients. ATS is a rare disease with broad phenotypic heterogeneity, making diagnosis challenging. Affected individuals typically present with episodes of periodic paralysis or cardiac symptoms in their first or second decade.
The ongoing clinical trials on Andersen tawil syndrome help the market to grow during the forecast period, as clinical trials help assess the safety and effectiveness of potential treatments for ATS. For instance, according to clinicaltrail.gov in September 2022 Centre Hospitalier Universitaire de Liege & University of Liege started a clinical study on Baby Detect Genomic Newborn Screening on which the study objective is to identify 126 treatable severe early onset genetic diseases at birth caused by 361 genes. These studies are basically for the conditions with, Congenital Adrenal Hyperplasia, Andersen Tawil Syndrome and others. Hence, this study is estimated to be completed by August 2025.
Complications Associated with Disease are one of the factors that hamper the market to grow market further. Hence Andersen Tawil syndrome (ATS) has various side effects, including periodic paralysis, which causes muscle weakness or paralysis, primarily in the legs, and can be triggered by exercise, stress, or hot weather. Arrhythmias, abnormal heart rhythms, can cause racing heartbeats, shortness of breath, or fainting, and can be life-threatening. Dysmorphic features, such as low-set ears, wide-spaced eyes, and short stature, can also result from ATS. These side effects vary depending on the severity of the condition.
The global Andersen-Tawil Syndrome market is segmented based on disease type, drug class, route of administration, distribution channel and region.
The Type 1 segment from the disease type segment accounted for 41.7% and it is expected to be dominated during the forecast period. Type 1 Atrial Syndrome (ATS) is caused by mutations in the KCNJ11 gene, affecting 1 in 100,000 to 1 in 1 million people. Symptoms typically appear in childhood or adulthood, with severity varying from person to person.
For instance, in November 2022 Centro Nacional de Investigations Cardiovascular (CNIC) discovered the cause of arrhythmias and sudden death in the rare disease Andersen-Tawil syndrome type 1 (ATS1), which is caused by mutations affecting potassium channels that regulate electrical activity and the intracellular calcium cycle in cardiac and skeletal muscle.
The teams are led by Drs. Jose Jalife and Juan Antonio Bernal have discovered a previously unknown function of Kir2.1 channels, which control the essential electrical properties of excitable cells such as cardiac muscle, skeletal muscle, and neurons.
North America is estimated to hold about 38.4 % of the total market share throughout the forecast period, due to the presence of major key players, advancement in treatment options, well-developed healthcare sector, rising prevalence of disease, and growing awareness towards health in this region. The improvement would be possible because of marketing examination instruments and because of existing players who have positively gotten comfortable with the market since the beginning.
For instance, in 2021, Novartis AG launched a new medication called KEVEYIS (dichlorphenamide) for the treatment of periodic paralysis, a symptom of Andersen-Tawil syndrome.
The major global players in the market include: Merck, Grevis Pharmaceuticals, Xeris Pharma, Novartis AG, Advanz Pharmaceuticals, Alembic Pharmaceuticals, Avet Pharmaceuticals, Hikma Pharmaceuticals, Micro Labs, Advagen Pharma and among others.
COVID-19 had a substantial impact on the Andersen Tawil Syndrome market and vaccination for patients with neuromuscular disorders evolved during this pandemic in patients having these disorders. Skeletal muscle channelopathies encompass a group of rare genetic neuromuscular conditions, including myotonia congenita (MC), sodium channel myotonia (SCM), paramyotonia congenita (PMC), hypokalemic periodic paralysis (hypoPP), hyperkalemic periodic paralysis (hyperPP), and Andersen-Tawil syndrome (ATS). These conditions are characterized by episodic symptoms ranging from myotonia to periodic paralysis caused by the dysfunction of specific ion channels expressed in skeletal muscle.
The Global Andersen-Tawil Syndrome Market report would provide approximately 61 tables, 58 figures and 186 Pages.
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