Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.
Inclusion Criteria:
Group A: Affected Individuals (Rare or Genetic Disease Group)
Suspected or confirmed diagnosis of a rare or genetic disease based on clinical evaluation, molecular testing, family history, or other relevant findings.
Ability to provide informed consent (or have a legally authorized representative provide consent if the participant is a minor or unable to consent).
Willingness to participate in study procedures which may include biospecimen collection, data sharing including clinical data for research purposes, and participation in questionnaires.
All ages and sexes are eligible, including pediatric and adult participants.
Group B: Unaffected Individuals (Control Group)
No known personal history of the rare or genetic disease under study, or of a closely related condition.
May be a biological family member (e.g., parent, sibling) or an unrelated control, as appropriate to the study design.
Ability to provide informed consent (or have a legally authorized representative provide consent).
Willingness to participate in study procedures which may include biospecimen collection, data sharing including clinical data for research purposes, and participation in questionnaires.
All ages and sexes are eligible, including pediatric and adult participants.
Exclusion Criteria:
Groups A and B (unless otherwise specified)
Inability or unwillingness to provide informed consent or assent, or lack of a legally authorized representative (if required).
Presence of a condition or situation that, in the opinion of the investigators, would make participation unsafe, impractical, or not in the best interest of the participant.
For the control group (Group B): any known diagnosis, symptoms, or genetic findings suggestive of a rare or inherited disease relevant to the study’s focus.
Note: Other protocol defined Inclusion/Exclusion Criteria may apply.
Eligibility last updated 08/11/2025. Questions regarding updates should be directed to the study team contact.