Mawakamten – pionierski inhibitor miozyny sercowej w terapii kardiomiopatii przerostowej

Autorzy

Mateusz Gołębiowski - ; Nina Jankowska; Tymoteusz Borowski; Maria Zimoń; Joanna Bogacz; Aleksandra Dziegciarz

Słowa kluczowe:

mawakamten, kardiomiopatia przerostowa, inhibitor miozyny sercowej

Streszczenie

Abstrakt:

 

Mawakamten, selektywny, allosteryczny i odwracalny inhibitor miozyny sercowej wprowadza przełomowe podejście w terapii kardiomiopatii przerostowej (ang. hypertrophic cardiomyopathy, HCM), zdecydowanie różniąc się od klasycznych terapii. Mawakamten  działa bezpośrednio na patofizjologię choroby precyzyjnie redukując nadmierną kurczliwość redukując ilość głów miozyny. Skutkuje to poprawą parametrów sercowych i krążeniowych u pacjentów, a także umożliwia cofanie patologicznego przerostu mięśnia sercowego. Dzięki temu może nie tylko poprawić funkcję serca ale również zapobiegać lub opóźniać konieczność interwencji chirurgicznych , stanowiąc obiecującą alternatywę w terapii HCM. Dodatkowo jego innowacyjny uchwyt działania otwiera możliwość stosowania leku i innych chorobach mięśnia sercowego o podobnym patomechanizmie.

 

Słowa kluczowe: mawakamten, kardiomiopatia przerostowa, inhibitor miozyny sercowej

 

Mavacamten, a selective, allosteric, and reversible cardiac myosin inhibitor, introduces a groundbreaking approach to the treatment of hypertrophic cardiomyopathy (HCM), markedly differing from conventional therapies. Mavacamten acts directly on the pathophysiological mechanism of the disease by precisely reducing excessive contractility through a decrease in the number of active myosin heads. This leads to an improvement in cardiac and hemodynamic parameters in patients and enables regression of pathological myocardial hypertrophy. As a result, it may not only improve cardiac function but also prevent or delay the need for surgical intervention, representing a promising alternative in HCM therapy. Moreover, its innovative mechanism of action opens the possibility for its application in other myocardial diseases sharing similar pathophysiological pathways.

 

Keywords: mavacamten, hypertrophic cardiomyopathy, cardiac myosin inhibitor

 

Bibliografia

Referencje

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Opublikowane

24 sierpnia 2025