Evicenter
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RP-A501 (AAV9.LAMP2B)

Danon disease

Regulatory submission
BLA planned for late 2028
Last updated
300 sources

Product information and disease descriptionPlace of product in therapyApproaches to treatmentCurrent treatment options and standard of care

Arrhythmia management

16 evidence topics · 13 sources

Consensus statement: electrophysiological study and ablation for pre-excitation

“One of the frequent ECG findings is pre-excitation or Wolff-Parkinson-White syndrome, which is observed in almost one-half of patients, sometimes preceding overt hypertrophy.” (opens the source at this quote in a new tab)

“consequently electrophysiological studies and ablation should be considered in patients with pre-excitation.” (opens the source at this quote in a new tab)

Consensus statement: rhythm monitoring for atrial fibrillation and atrioventricular block

“Frequent 24-h Holter monitoring (at least yearly or every 6 months if the left atrium is dilated) or even the consideration of implantable loop recorders is recommended based on the high incidence of atrial fibrillation and advanced atrioventricular bloc.” (opens the source at this quote in a new tab)

“The incidence of nonsustained ventricular arrhythmia and its role as a predictor of sudden cardiac death is unknown.” (opens the source at this quote in a new tab)

“In the experience of this panel, nonsustained ventricular tachycardia is not rare in Danon disease.” (opens the source at this quote in a new tab)

Pacemaker implantation for heart block or sinus node dysfunction

“High-degree heart block or sinus node dysfunction requiring pacemaker implantation is also highly prevalent and is reported to be between 35% and 50%.” (opens the source at this quote in a new tab)

“Bradyarrhythmias may either be present at diagnosis (sometimes concomitantly with pre-excitation) or evolve at a later stage of the disease.” (opens the source at this quote in a new tab)

Italian Society of Cardiology position paper: ablation recommendation that names Danon disease

“catheter ablation may be considered when symptoms cannot be relieved by pharmacological therapy alone considering the balance between clinical status and recurrence risk.” (opens the source at this quote in a new tab)

GeneReviews: electrophysiologic study, ablation, and reduced ablation efficacy

“Consideration of electrophysiologic studies & ablation therapy” (opens the source at this quote in a new tab)

“Efficacy of ablations for accessory pathways & arrhythmia foci may be ↓ in those w/Danon disease.” (opens the source at this quote in a new tab)

Ablation outcomes and repeat procedures in a United States registry

“Cardiac ablation has also achieved some success in eliminating arrhythmias in Danon disease patients” (opens the source at this quote in a new tab)

“however, anecdotally several patients in our registry have required multiple ablation procedures possibly reflecting diffuse and progressive fibrosis that is challenging to eliminate by ablation therapy.” (opens the source at this quote in a new tab)

Frequency of arrhythmias and ablation procedures in males

“Arrhythmias and cardiac ablation procedures are common in 53% and 41%, respectively.” (opens the source at this quote in a new tab)

Frequency of ablation procedures and defibrillator implantation in females

“Approximately a third of females received cardiac ablation procedures, 31% underwent defibrillator implantation.” (opens the source at this quote in a new tab)

Electrophysiological study findings with pre-excitation: fasciculoventricular and extranodal pathways in a multicenter cohort

“Thirteen of 40 patients (32.5%) with Danon disease were found to have preexcitation (mean age 17.3 years; 38% women).” (opens the source at this quote in a new tab)

“EPS performed in 9 of 13 patients (69%) demonstrated FVP only in 2 (22.2%), extranodal pathway without exclusion of FVP in 2 (22.2%), and both FVP and extranodal pathway in 5 (55.6%).” (opens the source at this quote in a new tab)

“Two patients had malignant accessory pathway (AP) properties.” (opens the source at this quote in a new tab)

“These findings suggest patients with preexcitation and Danon disease should undergo EPS to assess for FVP and potentially malignant extranodal AP.” (opens the source at this quote in a new tab)

Electrophysiological study findings with pre-excitation: fasciculoventricular pathways in two smaller series

“Preexcitation pattern on ECG was found in four (40%) patients.” (opens the source at this quote in a new tab)

“Of these, two underwent an EPS which confirmed the presence of an FVP.” (opens the source at this quote in a new tab)

“Four patients who underwent electrophysiological studies all had FVPs, and 2 of them still had left-side atrioventricular pathways.” (opens the source at this quote in a new tab)

Electrophysiological study findings: accessory pathway excluded in all 4 males of a three-family series

“However, the presence of an accessory pathway was excluded by an EP study in all patients.” (opens the source at this quote in a new tab)

“The present study indicates that the distinct surface ECG pattern in Danon disease is not associated with ventricular pre-excitation.” (opens the source at this quote in a new tab)

Atrial fibrillation and thromboembolic stroke

“Moreover, 5 of the 7 patients suffered from atrial fibrillation (AF), with 1 of them experiencing thromboembolic stroke at the age of 30 years.” (opens the source at this quote in a new tab)

“Atrial fibrillation is frequently observed in these patients and may be associated with thromboembolic events in the young, while sustained ventricular arrhythmias occur less frequently than previously reported.” (opens the source at this quote in a new tab)

“Paroxysmal or permanent atrial fibrillation was more common in females (55% vs. 27% in males, p=0.02).” (opens the source at this quote in a new tab)

Consensus statement: embolic stroke from intracardiac thrombus

“Embolic strokes from intracardiac thrombi, in the setting of depressed left ventricular function, apical aneurysms, or atrial fibrillation or flutter can also occur.” (opens the source at this quote in a new tab)

Anticoagulation: hypertrophic cardiomyopathy guideline text that does not name Danon disease

“Although no RCTs have been published, the risk of systemic embolization is high in patients with HCM with AF.” (opens the source at this quote in a new tab)

Anticoagulation after left ventricular apical thrombus and stroke: single case

“In the case of intracardiac thrombus in the left heart, aggressive anticoagulation should be initiated to prevent further thromboembolic events.” (opens the source at this quote in a new tab)

“There has been no recurrence of feeling of numbness, hemiplegia, or aphasia after she was placed on daily warfarin under strict supervision.” (opens the source at this quote in a new tab)

Catheter ablation of atrial fibrillation: single case

“Of note, AF ablation in Patient II.3 of the present study effectively restored the sinus rhythm and improved his general condition.” (opens the source at this quote in a new tab)

“However, whether aggressive treatment of AF in this setting favorably affects the evolution of heart failure remains elusive.” (opens the source at this quote in a new tab)