Cost-effectiveness analyses and utility weights
Published cost-effectiveness analyses and health-state utility weights that a model of this product could cite, adapt, or compare against, grouped by how each article relates to the product and its indication. Every value is quoted from the article it cites, and section 3 lists the searches that found the articles.
13 cost-effectiveness analyses and 6 studies reporting utility weights · 13 sources
Overview
| Item | Detail |
|---|---|
| Literature searched | Tufts Medical Center Cost-Effectiveness Analysis Registry, searched October 21, 2026; the queries are listed in section 3. |
| Cost-effectiveness analyses | 13 |
| Studies reporting utility weights | 6 |
Cost-effectiveness analyses
Analyses of RP-A501 (AAV9.LAMP2B)
No evidence found.
Treatments for the same indication
No evidence found.
The same disease in other populations or settings
No evidence found.
Related diseases and analogous treatments
Nusinersen and onasemnogene abeparvovec versus best supportive care in spinal muscular atrophy, United States: ICER (2019)
Relevance: Evaluates onasemnogene abeparvovec, a systemic AAV9 gene therapy listed among the price benchmarks in section 4.1, and nusinersen against best supportive care in spinal muscular atrophy from a United States health care sector perspective.
Etranacogene dezaparvovec versus factor IX prophylaxis in severe hemophilia B, United States: Sarker et al. (2025)
Relevance: Evaluates etranacogene dezaparvovec, a systemic AAV gene therapy listed among the price benchmarks in section 4.1, against factor IX prophylaxis in severe hemophilia B from a United States health system perspective.
“A semi-Markov model simulated a cohort of adult males with severe hemophilia B to assess the economic impact of EDZ versus FIX prophylaxis over a lifetime horizon from a health system perspective in the USA.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Scenario analyses incorporated frameworks suggested by the Institute for Clinical and Economic Review for single or short-term transformative therapies.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Base-case analysis showed that at a cost of US$3.5 million, EDZ led to lifetime cost savings of US$11 million and an additional 0.64 quality-adjusted life years (QALYs) compared with FIX.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“When annual cost offsets attributed to EDZ were capped at US$150,000, EDZ was found to have a threshold price of US$3.1 million at a willingness-to-pay of US$150,000 per QALY.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“EDZ proved to be a dominant strategy over FIX prophylaxis in the base-case scenario, providing large cost savings and slightly better outcomes.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Eladocagene exuparvovec versus best supportive care in aromatic L-amino acid decarboxylase deficiency, United States: Zhang et al. (2026)
Relevance: Evaluates eladocagene exuparvovec against best supportive care in aromatic L-amino acid decarboxylase deficiency from a modified United States societal perspective that includes quality-of-life impacts on parents and caregivers.
“To conduct a US, modified societal perspective cost-utility analysis comparing eladocagene exuparvovec versus best supportive care (BSC).” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Health-state utilities were from a US time-trade-off study that valued AADCd quality-of-life impacts.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The analysis was conducted from a modified US societal perspective. The modified perspective reflects quality-of-life impacts to parents and caregivers, beyond impacts on patients.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The model had a lifetime horizon and cycle length of 3 months (the shortest interval between visits in the clinical studies).” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Outcomes were discounted (3%); costs were reported in 2024 US dollars.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Total costs (Table 3 ) were higher for eladocagene exuparvovec ($4,352,439) compared with BSC ($207,566), corresponding to an incremental cost of $4,144,873 for eladocagene exuparvovec over BSC.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“In the base case, 24.60 LYs were achieved with eladocagene exuparvovec, an incremental LY gain of 11.85 compared with BSC.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The cost per QALY gained was $199,007” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Discounted incremental quality-adjusted life-years (QALYs) for eladocagene exuparvovec were 20.83 (multiples of the MSD of total PDMS-2) and 18.44 (motor milestones). Incremental cost per QALY ranged from $199,007–$224,104.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Voretigene neparvovec-rzyl versus standard care in RPE65-mediated inherited retinal disease, United States: Johnson et al. (2019)
Relevance: Evaluates voretigene neparvovec, a one-time AAV gene therapy whose launch price section 4.1 lists, against standard care in RPE65-mediated inherited retinal disease in the United States.
“In this economic analysis, a health state transition model based on visual acuity and field with a lifetime horizon was developed to estimate the cost-effectiveness of voretigene neparvovec-rzyl.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Indirect costs, including educational attainment, productivity, caregiver burden, and governmental programs, were estimated using published literature and data analysis of public national surveys.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Costs and benefits were discounted at 3.0% annually.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The model population included 70 patients with RPE65 -mediated IRD, with a mean age of 15 years; 42 of 70 patients (60%) were female.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Voretigene neparvovec-rzyl had higher QALYs (18.1 vs 8.6) and lower total lifetime costs ($2.2 million vs $2.8 million) than SC (ie, was dominant) when including indirect costs, resulting in cost-savings and QALY increases. When indirect costs were excluded, the cost per QALY was $79 618.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Voretigene neparvovec-rzyl remains cost-effective if at least 8.8% of the long-term treatment effect continues after year 3 when including indirect costs and 43.3% when excluding indirect costs, assuming a cost threshold of $150 000 per quality-adjusted life-year.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Alglucosidase alfa enzyme replacement therapy versus supportive therapy in classic-infantile Pompe disease, Netherlands: Kanters et al. (2014)
Relevance: Evaluates enzyme replacement therapy with alglucosidase alfa against supportive therapy in classic-infantile Pompe disease from a Dutch societal perspective; section 2 notes that Danon disease was originally termed glycogen storage disease type IIb because of histological similarities to Pompe disease.
“A patient simulation model was used to compare costs and effects of ERT with costs of effects of supportive therapy (ST).” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Quality of life was measured using the EQ-5D and assumed to be the same in both treatment groups.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Costs were calculated from a societal perspective. This implies that all costs are included, no matter to whom they accrue.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Costs were discounted at a rate of 4% and effects were discounted at a rate of 1.5% in accordance with Dutch guidelines for pharmacoeconomic research [ 22 ]. A lifetime time horizon was used and a cycle length of ½ year was used.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Cost components and associated unit costs (2009 prices)” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Lifetime incremental QALYs were 6.8. Incremental costs were estimated to be € 7.0 million, which primarily consisted of treatment costs (95%). The incremental costs per QALY were estimated to be € 1.0 million (range sensitivity analyses: € 0.3 million - € 1.3 million). The incremental cost per life year gained was estimated to be € 0.5 million.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Heart transplantation and destination left ventricular assist device versus inotrope-dependent medical therapy in end-stage heart failure, United States: Long et al. (2014)
Relevance: Compares heart transplantation and left ventricular assist device destination therapy with inotrope-dependent medical therapy for end-stage heart failure in the United States; mechanical circulatory support and heart transplantation are current treatment options for Danon disease listed in section 2.
“We developed a state-transition model to simulate 4 treatment options and associated morbidity and mortality. Transition probabilities, costs, and utilities were estimated from published sources.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“OHT with a median wait time of 5.6 months is estimated to increase life expectancy to 8.5 years, and costs <$100,000/quality-adjusted life-year gained, relative to inotrope-dependent medical therapy.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Bridge to transplant-LVAD followed by OHT further is estimated to increase life expectancy to 12.3 years, for $226,000/quality-adjusted life-year gained versus OHT.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Patients who instead received destination therapy-LVAD are estimated to live 4.4 years on average from extrapolation of recent constant hazard rates beyond the first year. This strategy costs $202,000/quality-adjusted life-year gained, relative to inotrope-dependent medical therapy.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Destination left ventricular assist device versus medical management in ambulatory advanced heart failure, United States: Baras Shreibati et al. (2017)
Relevance: Evaluates left ventricular assist devices against medical management in ambulatory advanced heart failure in the United States, the mechanical circulatory support option listed in section 2.
“We used these costs and efficacy data from published studies in a Markov model to project the incremental cost-effectiveness ratio (ICER) of destination LVAD therapy compared with that of medical management. We discounted costs and benefits at 3% annually and report costs as 2016 U.S. dollars.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“In the lifetime simulation model, LVAD increased quality-adjusted life-years (QALYs) (4.41 vs. 2.67, respectively), readmissions (13.03 vs. 6.35, respectively), and costs ($726,200 vs. $361,800, respectively) compared with medical management, yielding an ICER of $209,400 per QALY gained and $597,400 per life-year gained.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“These results were sensitive to LVAD readmission rates and outpatient care costs; the ICER would be $86,900 if these parameters were 50% lower.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Primary heart transplantation and re-transplantation in children, United States: Dayton et al. (2006)
Relevance: Evaluates primary heart transplantation and re-transplantation in children in the United States; heart transplantation is a current treatment option in section 2, and males with Danon disease progress to end-stage hypertrophic cardiomyopathy in the second or third decade of life.
“Data from 95 pediatric subjects undergoing transplantation at our institution from 1997 through 2004 were reviewed to determine the costs of pediatric heart transplantation. Costs included pre-transplant care, organ procurement, initial hospitalization and follow-up care. Life expectancy was derived from the United Network of Organ Sharing data set.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Data were reported as cost per quality-adjusted life-years (QALYs) gained, which were discounted at 3%.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Under base-case assumptions, costs per QALY gained were $49,679 for primary transplantation and $87,883 for re-transplantation.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Sensitivity analysis yielded a cost-utility range of $44,943 to $57,628 per QALY gained for primary transplantation and $70,834 to $103,661 per QALY gained for re-transplantation.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Prophylactic implantable cardioverter-defibrillator versus typical management in childhood dilated cardiomyopathy, United States: Feingold et al. (2010)
Relevance: Evaluates primary-prevention implantable cardioverter-defibrillators in children with dilated cardiomyopathy in the United States; defibrillator implantation is the first current treatment option listed in section 2.
“We developed a Markov model to compare typical management of childhood dilated cardiomyopathy with symptomatic heart failure to prophylactic ICD implantation plus typical management.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“a hypothetical cohort (age 10–15 years) with dilated cardiomyopathy and symptomatic heart failure receiving outpatient medical management with or without an ICD” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The model adhered to the recommendations of the Panel on Cost-Effectiveness in Health and Medicine by examining costs from a societal perspective and applying a 3% annual discount to both costs and health benefits.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Time horizon was up to 20 years from model entry.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“All costs were adjusted using the gross domestic product deflator method and reported in 2006 US dollars.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Total per patient costs were $433,000 for the ICD strategy and $355,000 for typical management. Quality adjusted survival was greater for the ICD strategy relative to typical management (6.78 years vs. 6.43 years). Under base-case assumptions, the incremental cost utility ratio for the ICD strategy, as compared to typical management, was $281,622 per quality-adjusted life year (QALY) gained.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The ICD strategy cost less than $100,000/QALY only when the annual probability of sudden death as an outpatient exceeded 13% or when the ICD improved the patient’s quality of life by a utility of ≥ 0.11.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Implantable cardioverter-defibrillator and amiodarone versus no therapy for primary prevention of sudden cardiac death in hypertrophic cardiomyopathy, Canada: You et al. (2007)
Relevance: Evaluates implantable cardioverter-defibrillators and amiodarone for primary prevention of sudden cardiac death in hypertrophic cardiomyopathy in Canada; section 2 states that management of Danon disease is extrapolated from hypertrophic cardiomyopathy guidelines.
“Using a Markov model, we compared the health benefits and cost-effectiveness of 3 strategies for the primary prevention of SCD: implantable cardioverter/defibrillator (ICD) insertion, amiodarone therapy, or no therapy. We modeled hypothetical cohorts of 45-year-old patients with HCM with no history of cardiac arrest but at significant risk of SCD (3%/y).” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Over a lifetime, compared with no therapy, ICD therapy increased quality-adjusted survival by 4.7 quality-adjusted life years (QALYs) at an additional cost of $142,800 ($30,000 per QALY), whereas amiodarone increased quality-adjusted survival by 2.8 QALYs at an additional cost of $104,900 ($37,300 per QALY).” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Compared with no therapy, ICD therapy would cost < $50,000 per QALY for patients (i) aged 25, with > or = 1 risk factors for SCD, and (ii) aged 45 or 65, with > or = 2 risk factors for SCD.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Sudden cardiac death risk thresholds for implantable cardioverter-defibrillator implantation versus observed practice in hypertrophic cardiomyopathy, United Kingdom: Green et al. (2024)
Relevance: Evaluates sudden cardiac death risk thresholds for defibrillator implantation in hypertrophic cardiomyopathy from a United Kingdom National Health Service provider perspective; section 2 records that these risk models are not validated in Danon disease.
“A discrete-time Markov model was used to determine the cost-effectiveness of different ICD decision-making rules for implantation. Several scenarios were investigated, including the reference scenario of implantation rates according to observed real-world practice.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The time horizon for simulated patient follow up was set at 12 years from time of ICD implant” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“A UK National Health Service (NHS) health service provider perspective was taken and only costs directly incurred by the NHS were included” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The discount rate used was the standard 3.5% for both costs and utilities.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Cox 5-year SCD risk >4% 3569 1670 7.73 −0.04 −38 616 −2750 Cox 5-year SCD risk >6% 1925 26 7.80 0.03 834 747” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“We found that for a 12-year time horizon and WTP threshold of £25 000 in the primary analysis, the decision rule in the observed data is more cost-effective than the 4% 5-year SCD risk threshold regardless of the size of budget since it is both more costly and more harmful in total (ICER −£38 616/QALY). The 6% SCD risk threshold is cost-effective (ICER £834/QALY) with up to approximately 70% probability and even at small WTP values.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Mavacamten with standard of care versus standard of care alone in obstructive hypertrophic cardiomyopathy, Netherlands: Wiethoff et al. (2026)
Relevance: Evaluates mavacamten with standard of care against standard of care alone in obstructive hypertrophic cardiomyopathy from a Dutch societal perspective, with health states defined by NYHA class.
“A Markov model was developed in R based on the Decision Analysis in R for Technologies in Health framework with data from the EXPLORER-HCM trial.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“For the model, four health states were defined based on the NYHA classes, including NYHA I-NYHA III/IV and death. The model evaluated mavacamten with SoC versus SoC alone over a lifetime horizon with a cycle length of 4 weeks, following the most recent Dutch guidelines.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The objective was to develop a decision analytic model to evaluate the cost effectiveness of mavacamten compared with SoC in oHCM patients from a Dutch societal perspective.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Results indicate mavacamten being more effective (Δ4.75 LYs; Δ3.36 QALYs) and more costly (Δ€235,951) compared with SoC with an ICER of €70,223 per QALY gained.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The probability of the intervention being cost effective at the willingness-to-pay thresholds €50,000 and €80,000 per QALY was 1.3% and 87.4%, respectively.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Proband genetic testing added to clinical screening versus clinical screening alone in families with hypertrophic cardiomyopathy, Australia: Ingles et al. (2012)
Relevance: Evaluates proband genetic testing added to periodic clinical screening in Australian families with hypertrophic cardiomyopathy; section 2 quotes the company's plan to multiply Danon disease diagnoses through the family cascade.
“A probabilistic Markov decision model was used to determine cost per quality-adjusted life-year and cost for each life-year gained when genetic testing is included in the management of Australian families with HCM, compared with the conventional approach of periodic clinical screening alone.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The incremental cost-effectiveness ratio (ICER) was $A785 (£510 or €587) per quality-adjusted life-year gained, and $A12 720 (£8261 or €9509) per additional life-year gained making genetic testing a very cost-effective strategy.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“As the cost of proband genetic testing decreased, the ICER decreased and was cost saving when the cost fell below $A248 (£161 or €185).” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Utility weights
Utility weights from analyses of RP-A501 (AAV9.LAMP2B)
No evidence found.
Utility weights for the same indication
No evidence found.
Utility weights for the same disease in other populations or settings
No evidence found.
Utility weights for related diseases and analogous treatments
Utility weights by NYHA class, obstructive hypertrophic cardiomyopathy: Wiethoff et al. (2026)
Relevance: EQ-5D-5L utilities by NYHA class in obstructive hypertrophic cardiomyopathy, derived from the AFFECT-HCM study.
Access: Not open access
Not quoted: The utility weights are reported in the full text, which is closed access.
“Health state utilities and societal costs were derived from the AFFECT-HCM study, with utilities measured using the EQ-5D-5L.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Varying parameters by 20% showed that the utility value of patients in NYHA class I (ICER: €57,199; €111,506 per QALY) and drug costs (ICER: €53,985; €86,555 per QALY) were most sensitive.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Utility weights for hypertrophic cardiomyopathy with and without an implantable cardioverter-defibrillator, hypertrophic cardiomyopathy: Green et al. (2024)
Relevance: Utilities for hypertrophic cardiomyopathy with and without an implantable cardioverter-defibrillator, with disutilities for implantation, implantation complications, and shocks, for the defibrillator therapy listed in section 2.
“The shock utility proportion is the ratio of shock utility and non-shock utility of 0.7/0.8” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Similarly, the manage with ICD proportion is 0.8/0.88, which is consistent with values found elsewhere.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Utility weights for clinical, borderline, and genotype-positive phenotype-negative hypertrophic cardiomyopathy, hypertrophic cardiomyopathy: Ingles et al. (2012)
Relevance: Utilities for clinical, borderline, and genotype-positive phenotype-negative hypertrophic cardiomyopathy, from a model of genetic testing in Australian families with the disease.
Access: Not open access
Not quoted: The utility weights are reported in the full text, which is closed access.
“A probabilistic Markov decision model was used to determine cost per quality-adjusted life-year and cost for each life-year gained when genetic testing is included in the management of Australian families with HCM” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Utility weights for inotrope-dependent medical therapy, left ventricular assist device support, heart transplantation, and complications, end-stage heart failure: Long et al. (2014)
Relevance: Utilities for inotrope-dependent medical therapy, left ventricular assist device support, heart transplantation, and their complications in end-stage heart failure, the advanced therapies section 2 lists for Danon disease.
Access: Not open access
Not quoted: The utility weights are reported in the full text, which is closed access.
Utility weights for non-inotrope-dependent heart failure, left ventricular assist device support, heart transplantation, and stroke, advanced heart failure: Baras Shreibati et al. (2017)
Relevance: Utilities for non-inotrope-dependent advanced heart failure, left ventricular assist device support, heart transplantation, and stroke, for the mechanical circulatory support and heart transplantation that section 2 lists.
Access: Not open access
Not quoted: The utility weights are reported in the full text, which is closed access.
Utility weights for outpatient dilated cardiomyopathy, heart transplantation, and defibrillator complications, childhood dilated cardiomyopathy: Feingold et al. (2010)
Relevance: Utilities for children with dilated cardiomyopathy, before and after heart transplantation, and disutilities for defibrillator complications, for the childhood and adolescent onset of cardiomyopathy in males with Danon disease.
“Our model assumed that one year of life as an outpatient with dilated cardiomyopathy and mildly, symptomatic heart failure equaled 0.71 year of ideal health. This estimate was based on data from adults with stable heart failure” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“We estimated a utility of 0.5 for children receiving inpatient care for decompensated heart failure” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Search strategy
| Dataset | Query | Results |
|---|---|---|
| Ratios | nusinersen OR risdiplam OR onasemnogene OR "spinal muscular atrophy" | 85 |
| Ratios | "cardiomyopathy" | 79 |
| Ratios | "ventricular assist" OR "heart transplant" OR "heart transplantation" | 93 |
| Ratios | "gene therapy" | 98 |
| Ratios | Pompe OR "glycogen storage" OR Fabry OR "lysosomal storage" | 21 |
| Ratios | mavacamten OR aficamten OR "hypertrophic cardiomyopathy" | 14 |
| Ratios | Danon OR LAMP2 OR "RP-A501" | 0 |
| Weights | "hypertrophic cardiomyopathy" OR Danon | 27 |
| Weights | "heart transplant" OR "heart transplantation" OR "ventricular assist" | 228 |
| Weights | "dilated cardiomyopathy" OR "pediatric cardiomyopathy" | 20 |
| Weights | "New York Heart Association" | 387 |
| Methods | Danon OR LAMP2 OR "RP-A501" OR "lysosome-associated membrane protein" | 0 |