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Hypertrophic cardiomyopathy (HCM) is the most common genetic cardiomyopathy worldwide and causes significant morbidity and mortality. For decades, medical treatment options have been limited and untargeted, with frequent need for invasive interventions not readily accessible to many HCM patients. More recently, our understanding of the genetic basis and pathophysiologic mechanism of HCM has grown significantly, leading to the discovery of a new class of medications, cardiac myosin inhibitors (CMIs), that shift myosin into the super-relaxed state to counteract the hypercontractility in HCM. Subsequent clinical trials have proven the mechanism and efficacy of CMIs in humans with obstructive HCM, and additional trials are under way in patients with nonobstructive HCM. With favourable results in the completed clinical trials and ongoing research on the horizon, CMIs represent a bright new era in the targeted management of HCM. This review is focused on the discovery of CMIs, provides a summary of the results of clinical trials to date, provides clinicians with a roadmap for implementing CMIs into practice, and identifies gaps in our current understanding as well as areas of ongoing investigation.
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http://dx.doi.org/10.1016/j.cjca.2024.01.022 | DOI Listing |
Stroke
September 2025
Department of Neurology, China National Clinical Research Center for Neurological Diseases, Beijing Tiantan Hospital, Capital Medical University.
Background: Risk stratification in posterior circulation ischemic stroke (PCIS) is challenging. Although the Posterior Circulation Ischemic Stroke Outcome Score (PCISOS) was developed to address this, its utility in minor PCIS and in identifying homogeneous populations for clinical trials or treatment-responsive subgroups remains uncertain.
Methods: CHANCE-2 (Clopidogrel in High-Risk Patients With Acute Non-disabling Cerebrovascular Events-II) was a multicenter, randomized trial that enrolled patients with minor stroke or high-risk transient ischemic attack who carried CYP2C19 loss-of-function alleles.
Dan Med J
August 2025
Department of Clinical Medicine, Aarhus University.
Introduction: Reverse total shoulder arthroplasty is a well-established treatment for patients with rotator cuff tear arthropathy. The outcome after reverse total shoulder arthroplasty has been investigated in several studies and national registries. However, the treatment has not been compared to non-surgical treatment.
View Article and Find Full Text PDFBiologics
September 2025
Department of Acupuncture and Moxibustion, Beijing Hospital of Traditional Chinese Medicine, Beijing, People's Republic of China.
Osteoarthritis (OA) is a prevalent chronic disease, characterized by progressive joint degeneration and primarily affects older adults. OA leads to reduced functional abilities, a lower quality of life, and an increased mortality rate. Currently, effective treatment options for OA are lacking.
View Article and Find Full Text PDFEur J Case Rep Intern Med
August 2025
General medicine department, Universidad de Cartagena, Cartagena, Colombia.
Background: Romosozumab is a sclerostin-inhibiting monoclonal antibody that is effective and safe for anabolic treatment in patients with osteoporosis. Its main adverse effects are local; the severity of these injection-site reactions in clinical trials was generally mild.
Case Report: We present a case of a 71-year-old Colombian woman with osteoporosis at very high risk of fractures with no relevant history of drug allergies.
Mol Ther Methods Clin Dev
June 2025
Shanghai Vitalgen BioPharma Co., Ltd., Shanghai 201210, China.
Bietti crystalline dystrophy (BCD) is an autosomal recessive disorder caused by loss-of-function mutations in the gene, characterized by crystal-like lipid deposits in the retina, progressive photoreceptor loss, and retinal pigment epithelium (RPE) deterioration. Currently, there are no approved treatments for BCD. VGR-R01, an investigational gene therapy, uses subretinal administration of recombinant adeno-associated virus type 8 (AAV8) vector to deliver the human CYP4V2 gene.
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