In 2019, South Carolina Governor Henry McMaster signed Dylan’s Law into effect. This law was passed in the wake of Dylan Emery’s death. The young boy had Krabbe disease, a…
Jacklyn and Derrick Shaw have supported each other through some of the toughest and most complicated situations that anyone could go through, from a rare disease diagnosis to the loss…
On September 28, 2022, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar. These webinar help provide updates to the rare disease community on legislation and other policy initiatives…
On August 25th, 2022, the American Society of Gene & Cell Therapy held a webinar as part of its Lunch and Learn series, which is intended to help people living…
Every day, Jackson Garwood’s parents, Darren and Rebecca, look at him and smile. There were points when they didn’t expect to have their son for this long. You see, Jackson…
According to a recent press release from Passage Bio, their investigational gene therapy for Krabbe disease, PBKR03, may be ready to move into the clinical trial stage of drug development.…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat rare or chronic illnesses. For the purpose of this designation, "rare illnesses" are defined…
Passage Bio has recently announced that Orphan Drug Designation has been granted by the European Commission for PBKR03, a gene therapy for Krabbe Disease. This condition has yet to have…
One of six-year-old Jackson Garwood's favorite things to do is listen to his father sing. Some of his favorite numbers include 'Talk Tonight' by Oasis and Aqualung's 'Brighter Than The…
The European Commission (EC) has granted the Orphan designation to Passage Bio for their Krabbe disease treatment, PBKR03. This adds another designation to the medication's list, as it has already…
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…
Patient-Focused Drug Development Meeting with the FDA Organized by the National Organization for Rare Disorders (NORD), this meeting will be a critical opportunity for patients to communicate directly with regulators…
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?…
The A Million Dreams Gala A Virtual Event This virtual gala is a valuable fundraiser event for KrabbeConnect, a nonprofit organization dedicated to supporting the Krabbe disease patient community and finding a…
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…
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…
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