Investigative Treatment for ENPP1 Deficiency Has Moved to Phase 1/2 Clinical Trial
Inozyme Pharma has recently announced that the first patient was dosed in its Phase 1/2 clinical trial of INZ-701 in adult patients with ABCC6 Deficiency
The Brighter Hope Foundation
Brighter Hope Foundation
The Brighter Hope Foundation is the only 501(c)(3) nonprofit organization dedicated to bettering the lives of
families impacted by GACI / ARHR2. Our mission is to connect families affected by
Generalized Arterial Calcification of Infancy or Hypophosphatemic Rickets caused by
ENPP1 or ABCC6 Deficiencies to each other and to the medical community. Our goal is to
provide information about what complications can occur as a result of this condition. The
organization strives to provide current educational resources and supports ongoing
research. Our community is small but growing, and we hope to offer support and
information to families affected by this disorder around the world.
Condition Awareness & Advocacy
Here is a list of conditions this partner raises awareness and advocacy for:
- Generalized Arterial Calcification of Infancy
- Hypophosphatemic Rickets
Patient Worthy Posts on Generalized Arterial Calcification of Infancy
Webinar: Hearing Loss, From GACI Global
On June 10, 2022, Patient Worthy partner GACI Global hosted an hour long webinar on the subject of hearing loss, featuring Dr. Karen Wilber. GACI
GACI/ARHR2 Awareness Day is February 1st: Spreading rare Disease Awareness
Generalized arterial calcification of infancy (GACI) and autosomal recessive hypophosphatemia rickets type 2 (ARHR2) Awareness Day is recognized each year on Feb 1. This is
World Orphan Drug Congress USA 2023: Inozyme HCP and Patient Engagement Executive Discuss Importance of Technology and Partnerships in Rare Disease Diagnosis and Treatment
The World Orphan Drug Congress USA 2023 was held from May 23-25, 2023, at the Gaylord National Resort & Convention Center in National Harbor, MD.
Rare Community Profiles: Inozyme’s Catherine Nester Discusses Newborn Screening and the GACI Diagnostic Delay
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease
Rare Community Profiles: Creating a Strong Patient Advocacy-Pharma Partnership: How Inozyme Pharma and GACI Global Came Together for Impact
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their
