November 2026 | Issue 4

Awareness Month in Action: 
Walk for a Cure Builds Momentum


Since launching Pediatric Cardiomyopathy Awareness Month in 2014, CCF has grown this September observance into a powerful movement to raise awareness of this rare, often underdiagnosed heart disease. 


This year, 13 Walk for a Cure events across the U.S. and abroad—including CCF’s first walk in Germany—raised more than $55,000 for family support programs.


CCF extended the campaign’s reach through educational content highlighting symptoms, the importance of family cardiac history, and the need for early recognition. Across social media, the campaign generated 83,600 views and impressions and attracted 129 new followers.

Thank you to everyone who walked, shared, and supported our community.



Learn more →

CCF Brings Family Perspectives to Rare Disease and Transplant Policy Discussions


NORD Rare Diseases & Orphan Products Breakthrough Summit

NYU Langone Health’s Transplant Ethics & Policy Conference

In October, CCF represented families affected by pediatric cardiomyopathy at two key meetings shaping rare disease care and heart transplantation.


Leah Mumm, CCF’s director of family and physician relations, joined more than 900 attendees at the National Organization for Rare Disorders (NORD®) Rare Diseases & Orphan Products Breakthrough Summit®. CCF is also collaborating with NORD to develop a Pediatric Cardiomyopathy Patient Registry to advance research.


Sheila Gibbons, CCF’s senior director of development, attended NYU Langone Health’s Transplant Ethics & Policy Conference to explore organ donation challenges and strategies to address shortages affecting children awaiting lifesaving heart transplants.


NORD Rare Diseases & Orphan Products Breakthrough Summit →

NYU Langone Health’s Transplant Ethics & Policy Conference →




CCF-Funded Research Advances Understanding of Pediatric Cardiomyopathy


The CCF-funded study, “Genotype-Phenotype Associations in Pediatric Hypertrophic Cardiomyopathy: A Study From the Pediatric Cardiomyopathy Registry,” published in the Journal of the American College of Cardiology (JACC): Heart Failure, found that children with more severe heart muscle thickening were often diagnosed younger and more likely to have a disease-causing MYBPC3 variant.


Read JACC: Heart Failure Publication →

Updates in pediatric cardiomyopathy treatment development


FDA Expands Camzyos Approval to Adolescents with Obstructive HCM

The FDA has approved Bristol Myers Squibb’s Camzyos® (mavacamten) for adolescents with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) who weigh at least 30 kg (66 lbs). The milestone follows years of research and clinical collaboration, including an adolescent clinical trial led in part by CCF Medical Advisors Dr. Joseph W. Rossano and Dr. Charles E. Canter. Children in the CCF community also participated. Read the announcement →


Tenaya Therapeutics: FDA Grants RMAT Designation to Investigational Gene Therapy

Tenaya's investigational gene therapy TN-401 received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC). Read the announcement →


Cytokinetics: New Data for Aficamten in Non-Obstructive HCM

Cytokinetics reported additional ACACIA-HCM study results for aficamten in non-obstructive HCM. Read the announcement →


Rocket Pharmaceuticals: FDA Alignment on Danon Disease Gene Therapy Study

Rocket Pharmaceuticals announced FDA alignment on the path to complete its Phase 2 study of RP-A501, an investigational gene therapy for Danon disease, a rare condition that can cause cardiomyopathy. Read the announcement →

Research Roundup is supported by Bristol Myers Squibb. The updates are independently selected by CCF and inclusion does not constitute an endorsement of any company’s products. 

 

Educational Materials Now Available in Arabic



CCF’s expert-reviewed educational materials are now available in Arabic, adding to existing English and Spanish resources. This expansion helps more families access trusted information to better understand pediatric cardiomyopathy and their child’s care.


مواد CCF التعليمية متاحة الآن باللغة العربية، مما يتيح للعائلات الناطقة بالعربية الوصول إلى معلومات موثوقة راجعها خبراء، لمساعدتهم على فهم اعتلال عضلة القلب لدى الأطفال ورعاية طفلهم بشكل أفضل.


Request Arabic Materials →


Explore our Experts on Pediatric Cardiomyopathy Series 


In CCF’s recent webinar, genetic counselor Cara Barnett, MS, CGC, explains when to consider genetic testing for pediatric cardiomyopathy (PCM), what to expect, and how to interpret results. Watch this and more expert-led webinars on CCF’s YouTube channel.


View Webinars →


CCF Names New Chair and Vice Chair of Medical Advisory Board

Joseph W. Rossano, MD, MS, MHCM

Shelley D. Miyamoto, MD

CCF is pleased to announce the appointment of Joseph W. Rossano, MD, MS, MHCM, as chair and Shelley D. Miyamoto, MD, as vice chair of its Medical Advisory Board. Dr. Rossano is chief of cardiology at Children’s Hospital of Philadelphia, and Dr. Miyamoto directs the cardiomyopathy program at Children’s Hospital Colorado. Their clinical expertise and research leadership will help shape CCF’s research and education priorities and advance its mission to improve the lives of children affected by cardiomyopathy.


Meet Our Advisors →

The Big Hearts Live Music Fundraiser at Thornton Distilling Co. in Illinois raised awareness of hypertrophic cardiomyopathy while honoring 16-year-old CCF Heart Kid Jaxson.


CCF corporate partner, Cytokinetics, held a staff Bake for a Cure fundraiser at their San Francisco, CA headquarters to raise funds in support of children with cardiomyopathy.


Long-time CCF family members, the Passavias, held a Halloween Bash in New York in honor of their 13-year- old son, Boden, who was diagnosed with hypertrophic cardiomyopathy.

Dick Yue

For his 90th birthday, Dick Yue honored the memory of his beloved grandchildren, Bryan and Kevin Yu, who died from hypertrophic cardiomyopathy (HCM). In lieu of gifts, he asked family and friends to donate to CCF, raising more than $8,000.


Family and friends also paid tribute to Dick’s lifelong love of travel. A retired civil engineer, he traveled the world training engineers in 20 countries.



When Nolan was diagnosed with cardiomyopathy at five months old, his parents faced an unfamiliar diagnosis and an uncertain future.


Learn more about Nolan’s story in our annual appeal letter being sent this month.


Please consider making a year-end gift. Your generosity helps more families find the support and resources they need so they can feel less alone.


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