About PRKAG2 Cardiomyopathy (AKA PRKAG2 Syndrome)
PRKAG2 (Protein Kinase AMP-activated non-catalytic subunit Gamma 2) Syndrome is an autosomal dominant, progressive cardiac disease caused by a mutation on the PRKAG2 gene that results in increased AMP-activated protein kinase (AMPK) activity. This causes excessive cardiac glycogen storage in the heart, leading to cardiac hypertrophy (thickening of heart muscle) and multiple different types of arrhythmias (irregular heartbeats), including Wolff-Parkinson-White syndrome, atrial fibrillation, and cardiac conduction system disease. As a result, patients with PRKAG2 syndrome are at an increased risk of experiencing sudden cardiac arrest or heart failure. There are no FDA-approved disease-modifying therapies for PRKAG2 Syndrome, and current standard of care focuses on symptom management with medications or invasive treatments including pacemakers or implantable cardioverter defibrillator (ICD). Patients who develop progressive heart failure may require a heart transplant.

About Avidity Biosciences
Avidity Biosciences, Inc.'s mission is to profoundly improve people's lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCs™). Avidity is revolutionizing the field of RNA with its proprietary AOCs, which are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to address targets and diseases previously unreachable with existing RNA therapies. Utilizing its proprietary AOC platform, Avidity demonstrated the first-ever successful targeted delivery of RNA into muscle and is leading the field with clinical development programs for three rare neuromuscular diseases: myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). Avidity is also advancing two wholly-owned precision cardiology development candidates addressing rare genetic cardiomyopathies. In addition, Avidity is broadening the reach of AOCs with its advancing and expanding pipeline including programs in cardiology and immunology through key partnerships. Avidity is headquartered in San Diego, CA.

About Citizen Health.  Citizen Health is a conumer health platform dedicated to leveraging technology and real-world data to enhance patient outcomes and streamline drug development. By securely providing patients access to medical records and supportive tools to address daily health challenges, Citizen Health fosters active patient engagement, benefiting both individuals and therapeutic innovation.

About Digital Natural History Studies A digital natural history study is a type of natural history study that uses data already collected in electronic medical records to observe how a disease progresses over time — without intervening or treating differently. A natural history study is a type of research that follows people over time who have a specific disease or condition, without trying to treat or change the course of the condition. The goal is to understand how the disease develops and progresses naturally — hence the name "natural history."

Key features of a natural history study: Observational: No experimental treatment is given — researchers just observe what happens.Longitudinal: It usually tracks participants over a long period to capture how symptoms change or evolve.
Detailed Data Collection: Includes information like genetics, biomarkers, symptoms, imaging, etc.
Helps Identify Patterns: Can reveal different stages of a disease, risk factors, or how it affects various people.
Why are natural history studies important? They provide a baseline for how a disease normally progresses. They help design better clinical trials by identifying the best time to intervene and what outcomes to measure. Especially valuable for rare diseases, where little is known and treatment options are limited.