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
KrabbeConnect’s mission is to be the source of comprehensive information and access to resources for patients with Krabbe disease. The foundation will drive state of the art research by bridging the gap between science and patient knowledge.
KrabbeConnect seeks to revolutionize the practice of medicine by identifying, optimizing, and implementing advances in the care and cure of globoid cell leukodystrophy.
Our dedication and efforts will advance research, provide family support, enhance education, and raise awareness, one step at a time. The organization will encourage patients, caregivers, medical scientists, clinicians, industry, and the government to work together quickly to assure a better life for patients. KrabbeConnect believes advances are made when we align ourselves with the same goal, improving the quality of life of those diagnosed with Krabbe disease.
We encourage you to learn more about us by understanding our Mission, Vision, and Multi-Center Approach here. If you have questions or want to connect with us, please Contact Us. We would love to hear from you!
Condition Awareness & Advocacy
Here is a list of conditions this partner raises awareness and advocacy for:
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Patient Worthy Posts on Krabbe Disease
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
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
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
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