Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Tenaya Therapeutics
Age
0–18
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Retrospective
Data is available for patient \<18 years of age. Patients must be \<18 years of age at enrollment or at time of death.
Documented results of genotyping showing the presence of at least one pathogenic or likely pathogenic MYBPC3 mutation (heterozygous, homozygous, or compound heterozygous).
For Infants:
Infants who are homozygous or compound heterozygous for the known pathogenic truncating MYBPC3 mutations are eligible.
For all other participants:
Age \<18 at entry into the prospective study.
Documented results of genotyping identifying at least one pathogenic or likely pathogenic MYBPC3 mutation (heterozygous, homozygous, or compound heterozygous).
Diagnosis of Cardiomyopathy (CM): HCM, DCM, RCM, mixed CM, or LVNC.
You may not be if
Patient received cardiac transplantation or died \>10 years before study initiation. For homozygous or biallelic infants, data may be collected beyond this 10-year period.
Prospective
Concurrent participation in an interventional clinical trial unless approved by the sponsor.
Severe noncardiac disease anticipated to significantly reduce life expectancy.
Clareo Health | Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy
