In October, I sat down with Laura and Ryan Nitahara to discuss their experience with Krabbe disease, a rare genetic lysosomal storage disorder and progressive neurological condition. Prompted by their…
The FDA has recently cleared an Investigational New Drug (IND) application for FBX-101, allowing it to move into a Phase 1/2 clinical trial. This gene therapy was developed for the…
During the drug development process, drug manufacturers must seek out an Investigational New Drug application (IND), which allows the treatment to be transported across state lines. IND clearance also…
As of last month, a newborn screening advisory committee from the Virginia Department of Health declined to add Krabbe disease to the conditions that doctors look for when a child…
A new treatment for Krabbe disease, PBKR03, has recently been granted the Orphan Drug and Rare Pediatric Disease designations by the FDA, according to an article in GlobeNewswire. These designations…
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?…
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…
According to a story from The Garstang Courier, Joshua Brandwood of Lancaster, UK has begun organizing a 5K run as a fundraiser for the family of Lexi Tindale, a girl…
As reported in Stem Cells News; finding the right donor for people in need of bone marrow transplants can be very tricky. This is because finding the correct match between…
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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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…
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…
Passage Bio has just recently announced that Krabbe disease will be the focus of their third clinical trial program. Krabbe Disease Krabbe disease is a rare, inherited, neurodegenerative lysosomal storage…
September is Krabbe Disease Awareness Month! Because a lot of times, these rare diseases are rarely talked about, it'll be useful to deep dive into the disease to learn 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…
Losing a child has to be one of the most heart-wrenching experiences a family can encounter, yet losing a child when it could have been prevented is even more devastating.…
In Chicago, a panel of lawmakers and parents are fighting back after the loss of children to a rare disease. Shermane Jenkins lost her two-year-old son to the very rare…
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