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Daily Archives: July 22, 2022

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Child with Aplastic Anemia Needs Stem Cell Donor
Source: Pixabay.com

Child with Aplastic Anemia Needs Stem Cell Donor

  • Post author:Jessica Lynn
  • Post published:July 22, 2022
  • Post category:Aplastic anemia

  To Diem Nguyen, her daughter Minh is her whole world. Diem would stop at nothing to ensure her daughter gets the help that she needs. That's why, recently, Diem…

Continue Reading Child with Aplastic Anemia Needs Stem Cell Donor
Insurance Finally Approves Surgery for Father with Pseudomyxoma Peritonei
source: pixabay.com

Insurance Finally Approves Surgery for Father with Pseudomyxoma Peritonei

  • Post author:Jessica Lynn
  • Post published:July 22, 2022
  • Post category:Pseudomyxoma peritonei

  In 2020, Anthony Di Laura and Jackie Cucullo were over-the-moon. The married couple had just learned that Jackie was pregnant with their first child. Anthony and Jackie were thrilled…

Continue Reading Insurance Finally Approves Surgery for Father with Pseudomyxoma Peritonei
The Strength of Rare: Life as a Koolen-De Vries Syndrome Patient, Parent, and Caregiver, Part 2

The Strength of Rare: Life as a Koolen-De Vries Syndrome Patient, Parent, and Caregiver, Part 2

  • Post author:Becky Tilley
  • Post published:July 22, 2022
  • Post category:Koolen-de Vries

Continued From Part One Written by Becky Tilley Finally I found a role that I was appreciated in, at least by the kids. I wasn't very welcome by the girls…

Continue Reading The Strength of Rare: Life as a Koolen-De Vries Syndrome Patient, Parent, and Caregiver, Part 2
Researchers Discover a Therapeutic Target for Wiskott-Aldrich Syndrome

Researchers Discover a Therapeutic Target for Wiskott-Aldrich Syndrome

  • Post author:Rose Duesterwald
  • Post published:July 22, 2022
  • Post category:Wiskott-Aldrich Syndrome

  Scientists have known for years that Wiskott-Aldrich syndrome (WAS) is caused by a gene on the X chromosome. The gene (the WASP gene) can be found throughout the immune…

Continue Reading Researchers Discover a Therapeutic Target for Wiskott-Aldrich Syndrome
Rare Classroom: Adult Polyglucosan Body Disease
source: shutterstock

Rare Classroom: Adult Polyglucosan Body Disease

  • Post author:James Moore
  • Post published:July 22, 2022
  • Post category:Adult Polyglucosan Body Disease (APBD)

Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…

Continue Reading Rare Classroom: Adult Polyglucosan Body Disease
Positive Data Available on AZP-3601 for Hypoparathyroidism
HeungSoon / Pixabay

Positive Data Available on AZP-3601 for Hypoparathyroidism

  • Post author:Jessica Lynn
  • Post published:July 22, 2022
  • Post category:hypoparathyroidism

In a recent news release, clinical-stage biotechnology company Amolyt Pharma ("Amolyt") shared that positive data was available from a proof-of-concept study evaluating AZP-3601 for hypoparathyroidism. Within this study, researchers sought…

Continue Reading Positive Data Available on AZP-3601 for Hypoparathyroidism

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