The study is a collaborative research effort among industry (Ultragenyx Pharmaceutical, Inc.), academia (Yale University), and a patient-advocacy group (The XLH Network, Inc.) to characterize XLH and other chronic hypophosphatemic disorders from the patient perspective. Research data is collected via a mobile/web-based platform, called Rarify™, in which participants answer periodic questions.
Promote long-term engagement of patients and Health Care Providers (HCPs). Aid efforts to educate newly diagnosed patients and their families.
Evaluate long-term progression of the disorder and increase understanding of hypophosphatemic disorders (including XLH and others).
Provide real-world data on health care resources and how they are used.
BEYONDXLH has gathered information from people with XLH and their caregivers since 2019. While the study is not currently enrolling new participants, new data from enrolled participants is constantly being added to this rich repository which we hope will begin to tell a more holistic story of what it’s like to live with XLH and other chronic hypophosphatemic disorders.
We call this area of beyondxlh.org the "BEYONDXLH Data Portal". The Data Portal is a password protected section available only to enrolled participants.
Participant data is very carefully managed in the web-based system including the use of third-party security vendors. One important aspect of this security is ensuring that the identity of participants/caregivers is unknown to Ultragenyx and its affiliates through the use of a technology known as ‘tokenization’. This means that personally identifying information is replaced by a unique study participant identification number. Only the highly qualified research team at Yale knows the true identity of the participants.
This study is not enrolling new participants at this time. If you are interested in future enrollment or for more information about this study you are invited to contact the research team at Yale by email at [email protected] .