Sir’Savion’s Story
Editor's Note: Patient Worthy is honored to share this article from our friends at the Brighter Hope Foundation. To see the article in its original format, please click here. Sir’Savion…
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Sir’Savion’s Story
Too Little Too Late: A Mom’s Plea for Improved GACI Screening
Acknowledgement: Patient Worthy is honored to share this story from our friends at GACI Global. To view the original article, please click here. Sharon’s passion for children shines in her…
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Too Little Too Late: A Mom’s Plea for Improved GACI Screening
The story of Laura and Martina and a rare disease called GACI with two very different outcomes.
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…
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The story of Laura and Martina and a rare disease called GACI with two very different outcomes.
ICYMI: INZ-701 Begets Positive Data in Clinical Study on ABCC6 Deficiency and ENPP1 Deficiency
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…
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ICYMI: INZ-701 Begets Positive Data in Clinical Study on ABCC6 Deficiency and ENPP1 Deficiency
Photo courtesy of GACI Global
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 families, advocates, scientists, and more.…
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Rare Community Profiles: Creating a Strong Patient Advocacy-Pharma Partnership: How Inozyme Pharma and GACI Global Came Together for Impact
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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. This conference is focused solely…
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World Orphan Drug Congress USA 2023: Inozyme HCP and Patient Engagement Executive Discuss Importance of Technology and Partnerships in Rare Disease Diagnosis and Treatment
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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 a time for spreading awareness…
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GACI/ARHR2 Awareness Day is February 1st: Spreading rare Disease Awareness
GACI Global’s Worldwide Walk
GACI Global's Worldwide Walk September 25, 2022 The GACI/ARHR2 community, their family, friends, and supporters from around the world will be unified on one day, around one shared cause, when…
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GACI Global’s Worldwide Walk
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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 Global is a nonprofit organization…
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Webinar: Hearing Loss, From GACI Global
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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 and the second cohort in…
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Investigative Treatment for ENPP1 Deficiency Has Moved to Phase 1/2 Clinical Trial
