Raising Money for Krabbe Disease Research: Quinton’s Quest for a Cure
Quinton's Quest for a Cure (photo submitted by Quinton's mother, Laura Nitahara)

Raising Money for Krabbe Disease Research: Quinton’s Quest for a Cure

When their son Quinton was diagnosed with late-onset Krabbe disease through newborn screening, Laura and Ryan Nitahara were initially full of questions. What was Krabbe disease? How would this affect their family?…

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New Reduced-Intensity Conditioning for HSCT Found Beneficial for 20 Rare Diseases

A recent study published in the journal Blood Advances has documented that hematopoietic stem cell transplantation (HSCT) is both safe and effective for children with many different kinds of inherited nonmalignant conditions…

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Bone Marrow Transplants and GALC: Learning to Treat Krabbe Disease
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Bone Marrow Transplants and GALC: Learning to Treat Krabbe Disease

A team of researchers recently clarified how cellular mechanisms work in the treatment of Krabbe disease. Previously, researchers believed that bone marrow transplants assisted in providing positive patient outcomes through…

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Found: Pelizaeus-Merzbacher Disease, the Cause, and a Potential Cure

  According to a recent article published by the University of California San Francisco, Pelizaeus-Merzbacher disease (PMD) is a rare neurological disease affecting young boys. PMD fatalities may occur before the child…

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2019 United Leukodystrophy Conference Highlights

Patient Worthy had the privilege of attending another United Leukodystrophy Conference. The ULF’s 2019 event took place at the end of June in Illinois and had a record setting number…

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Could This Mouth Swab Save Lives? Genepath Test Diagnosis Krabbe Disease and Much More
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Could This Mouth Swab Save Lives? Genepath Test Diagnosis Krabbe Disease and Much More

Ashley Wibberly’s son Levi was born in September of 2013 and lived for just 20 months with Krabbe disease. Ashley couldn’t have seen it coming. Now, however, a new test…

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