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市場調査レポート
商品コード
1345445
アンデルセン・タウィル症候群の世界市場-2023年~2030年Global Andersen-Tawil Syndrome Market - 2023-2030 |
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カスタマイズ可能
適宜更新あり
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アンデルセン・タウィル症候群の世界市場-2023年~2030年 |
出版日: 2023年09月06日
発行: DataM Intelligence
ページ情報: 英文 186 Pages
納期: 即日から翌営業日
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概要
アンデルセン・タウィル症候群の世界市場は、2022年に19億米ドルに達し、2023-2030年の予測期間中にCAGR 7.4%で成長し、2030年には33億米ドルに達すると予測されています。
アンデルセン・タウィル症候群は、KCNJ2遺伝子の変異によって引き起こされる遺伝性疾患です。この遺伝子は、心筋細胞からカリウムイオンを輸送するイオンチャネルをコードしています。このイオンチャネルは骨格筋と心筋の正常な機能を維持するために不可欠です。通常、常染色体優性遺伝で親から受け継がれますが、新たな遺伝子変異により発症することもあります。具体的な症状や重症度は、同じ家族であっても人によって大きく異なります。
さらに、世界のアンデルセン・タウィル症候群市場は、アンデルセン・タウィル症候群の有病率の増加、希少疾患の治療薬を優先する政府の関心の高まり、治療を支援し認知度を高めるための団体の参入の増加、メドトロニック社、アボット・ラボラトリーズ社、ボストン・サイエンティフィック社、リバノバ社などの主要企業を有する主要企業による研究開発投資の増加など、さまざまな要因によって牽引されています。
ダイナミクス
アンデルセン・タウィル症候群の有病率の上昇
アンデルセン・タウィル症候群の有病率の増加は、予測期間中に市場が成長するのに役立つ重要な要因の1つです。アンデルセン・タウィル症候群(ATS)は、再発性の弛緩性筋力低下、心不整脈、特徴的な骨格と顔貌を伴うまれな常染色体優性疾患です。有病率は1,000,000人に0.8~1人と推定されていますが、特定の集団およびATS患者のサブグループからの有病率の報告により、実際の有病率はより高いと予想されます。ATSは表現型の多様性をもつまれな疾患であり、診断が困難です。罹患者は通常、1~2歳代に周期性麻痺または心臓症状のエピソードを呈します。
アンデルセン・タウィル症候群に関する進行中の臨床試験
アンデルセン・タウィル症候群に関する臨床試験が進行中であることは、ATSに対する潜在的な治療法の安全性と有効性を評価するのに役立つため、予測期間中に市場が成長するのに役立ちます。例えば、clinicaltrail.govによると、2022年9月にCentre Hospitalier Universitaire de Liege &University of Liegeは、Baby Detect Genomic Newborn Screeningに関する臨床研究を開始しました。この研究目的は、361の遺伝子に起因する126の治療可能な重症早期発症遺伝病を出生時に特定することです。これらの研究は基本的に、先天性副腎過形成、アンデルセン・タウィル症候群などを対象としています。したがって、この研究は2025年8月までに完了する予定です。
疾患に伴う合併症
疾患に伴う合併症は、市場のさらなる成長を妨げる要因の1つです。アンデルセン・タウィル症候群(ATS)には、主に脚の筋力低下や麻痺を引き起こす周期性麻痺など、さまざまな副作用があり、運動やストレス、暑い気候などが引き金となります。不整脈(異常な心臓のリズム)は、動悸、息切れ、失神を引き起こし、生命を脅かすことがあります。耳の位置が低い、目の間隔が広い、背が低いなどの異形性も、ATSの影響によるものです。これらの副作用は症状の重症度によって異なります。
このレポートの詳細サンプル請求
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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