Only about 50% of those born with GACI survive. Patient Worthy is grateful to GACIGlobal for the ability to share Sienna's Story. GACI Global is a nonprofit organization whose mission…
This article is written by Enrico Orzes and originally published on Osservatorio Malattie Rare (OMaR). It has been translated from Italian. Patient Worthy is grateful to the team at OMaR…
Biopharmaceutical company Inozyme Pharma ("Inozyme") has become a leader at the forefront of developing innovative therapies for rare diseases such as ENPP1 deficiency (the childhood form is known as generalized…
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 families, advocates, scientists, and more.…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
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. This conference is focused solely…
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 a time for spreading awareness…
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 Global is a nonprofit organization…
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 and the second cohort in…
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